Trial Outcomes & Findings for Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria (NCT NCT05270837)
NCT ID: NCT05270837
Last Updated: 2026-07-21
Results Overview
The blood Phe measurement following 72 weeks on study is defined as the average of the Week 69 and Week 73 blood Phe measurements. Change from baseline in blood Phe will be compared between participants in the active (pegvaliase) and the control (diet-only) treatment arms. Baseline blood Phe concentration at Part 1 was defined as the average of 3 pre-treatment measurements collected between Screening/Run-in (2) and Day 1 pre-dose (1).
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE3
Target enrollment
55 participants
Primary outcome timeframe
Baseline to Week 72 (average of the Week 69 and Week 73)
Results posted on
2026-07-21
Participant Flow
This study was conducted by 13 principal investigators at 13 study centers in the United States of America and 3 principal investigators at 3 study centers in Germany.
Approximately 54 participants planned (36 pegvaliase; 18 diet-only); 55 participants randomized (36 pegvaliase; 19 diet-only) and 49 participants completed Part 1 (32 pegvaliase; 17 diet-only); the other 6 participants discontinued from study before reaching Week 73.
Participant milestones
| Measure |
Experimental: Pegvaliase (Part 1 and Part 2)
Pegvaliase (Part 1 and Part 2): Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only (Part 1) Then Pegvaliase Treatment (Part 2)
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72 (Part 1).
From Week 73 (Part 2): Initiate pegvaliase treatment to follow the same schedule for dosing, study visits and assessments as the pegvaliase arm participants
|
|---|---|---|
|
Part 1 (Through Week 72)
STARTED
|
36
|
19
|
|
Part 1 (Through Week 72)
COMPLETED
|
32
|
17
|
|
Part 1 (Through Week 72)
NOT COMPLETED
|
4
|
2
|
|
Part 2 (Week 73 Onwards)
STARTED
|
32
|
16
|
|
Part 2 (Week 73 Onwards)
Completed Part 1, Did Not Enter Part 2, Continuing in Study
|
0
|
1
|
|
Part 2 (Week 73 Onwards)
COMPLETED
|
0
|
0
|
|
Part 2 (Week 73 Onwards)
NOT COMPLETED
|
32
|
16
|
Reasons for withdrawal
| Measure |
Experimental: Pegvaliase (Part 1 and Part 2)
Pegvaliase (Part 1 and Part 2): Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only (Part 1) Then Pegvaliase Treatment (Part 2)
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72 (Part 1).
From Week 73 (Part 2): Initiate pegvaliase treatment to follow the same schedule for dosing, study visits and assessments as the pegvaliase arm participants
|
|---|---|---|
|
Part 1 (Through Week 72)
Adverse Event
|
1
|
0
|
|
Part 1 (Through Week 72)
Physician Decision
|
1
|
0
|
|
Part 1 (Through Week 72)
Withdrawal by Subject
|
2
|
2
|
|
Part 2 (Week 73 Onwards)
Withdrawal by Subject
|
1
|
0
|
|
Part 2 (Week 73 Onwards)
Adverse Event
|
0
|
1
|
|
Part 2 (Week 73 Onwards)
Continuing in Study
|
31
|
15
|
Baseline Characteristics
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
Baseline characteristics by cohort
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=19 Participants
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
Total
n=55 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
14 years
|
12 Participants
n=9 Participants
|
7 Participants
n=27 Participants
|
19 Participants
n=267 Participants
|
|
Age, Continuous
|
14.4 years
STANDARD_DEVIATION 1.27 • n=9 Participants
|
14.1 years
STANDARD_DEVIATION 1.29 • n=27 Participants
|
14.3 years
STANDARD_DEVIATION 1.27 • n=267 Participants
|
|
Age, Customized
12 years
|
3 Participants
n=9 Participants
|
4 Participants
n=27 Participants
|
7 Participants
n=267 Participants
|
|
Age, Customized
13 years
|
5 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
5 Participants
n=267 Participants
|
|
Age, Customized
15 years
|
10 Participants
n=9 Participants
|
6 Participants
n=27 Participants
|
16 Participants
n=267 Participants
|
|
Age, Customized
16 years
|
4 Participants
n=9 Participants
|
2 Participants
n=27 Participants
|
6 Participants
n=267 Participants
|
|
Age, Customized
17 years
|
2 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=9 Participants
|
11 Participants
n=27 Participants
|
33 Participants
n=267 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=9 Participants
|
8 Participants
n=27 Participants
|
22 Participants
n=267 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=9 Participants
|
19 Participants
n=27 Participants
|
53 Participants
n=267 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
2 Participants
n=267 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=9 Participants
|
18 Participants
n=27 Participants
|
48 Participants
n=267 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=267 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=9 Participants
|
1 Participants
n=27 Participants
|
4 Participants
n=267 Participants
|
|
Region of Enrollment
United States
|
28 participants
n=9 Participants
|
16 participants
n=27 Participants
|
44 participants
n=267 Participants
|
|
Region of Enrollment
Germany
|
8 participants
n=9 Participants
|
3 participants
n=27 Participants
|
11 participants
n=267 Participants
|
|
BMI
|
24.9 kg/m^2
STANDARD_DEVIATION 5.43 • n=9 Participants
|
24.0 kg/m^2
STANDARD_DEVIATION 6.33 • n=27 Participants
|
24.6 kg/m^2
STANDARD_DEVIATION 5.71 • n=267 Participants
|
|
Blood Phe Concentration
|
1025.3 µmol/L
STANDARD_DEVIATION 254.05 • n=9 Participants
|
1028.6 µmol/L
STANDARD_DEVIATION 199.39 • n=27 Participants
|
1026.4 µmol/L
STANDARD_DEVIATION 234.70 • n=267 Participants
|
|
Total protein intake
|
1.1 g/kg
STANDARD_DEVIATION 0.37 • n=9 Participants
|
1.1 g/kg
STANDARD_DEVIATION 0.41 • n=27 Participants
|
1.1 g/kg
STANDARD_DEVIATION 0.38 • n=267 Participants
|
|
Protein from intact food
|
0.3 g/kg
STANDARD_DEVIATION 0.20 • n=9 Participants
|
0.2 g/kg
STANDARD_DEVIATION 0.15 • n=27 Participants
|
0.3 g/kg
STANDARD_DEVIATION 0.18 • n=267 Participants
|
|
Protein from medical food
|
0.8 g/kg
STANDARD_DEVIATION 0.33 • n=9 Participants
|
0.9 g/kg
STANDARD_DEVIATION 0.35 • n=27 Participants
|
0.8 g/kg
STANDARD_DEVIATION 0.34 • n=267 Participants
|
|
PAL IgG
|
8 Participants
n=9 Participants
|
3 Participants
n=27 Participants
|
11 Participants
n=267 Participants
|
|
PEG IgG
|
20 Participants
n=9 Participants
|
6 Participants
n=27 Participants
|
26 Participants
n=267 Participants
|
|
PAL IgM
|
18 Participants
n=9 Participants
|
5 Participants
n=27 Participants
|
23 Participants
n=267 Participants
|
|
PEG IgM
|
36 Participants
n=9 Participants
|
10 Participants
n=27 Participants
|
46 Participants
n=267 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 72 (average of the Week 69 and Week 73)Population: The Intent to Treat (ITT) population will consist of all participants who are randomized. Participants will be analyzed according to the randomized treatment for the analysis of efficacy.
The blood Phe measurement following 72 weeks on study is defined as the average of the Week 69 and Week 73 blood Phe measurements. Change from baseline in blood Phe will be compared between participants in the active (pegvaliase) and the control (diet-only) treatment arms. Baseline blood Phe concentration at Part 1 was defined as the average of 3 pre-treatment measurements collected between Screening/Run-in (2) and Day 1 pre-dose (1).
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=19 Participants
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Change From Baseline in Blood Phe Concentration Following 72 Weeks (Average of the Week 69 and Week 73) on Study.
|
-449.9 µmol/L
Standard Deviation 292.33
|
-34.4 µmol/L
Standard Deviation 272.07
|
PRIMARY outcome
Timeframe: Up to Week 73Population: Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
Treatment-emergent adverse events (TEAE) is defined as any AE that newly appeared or worsened in severity after first dose of pegvaliase (pegvaliase arm) or on or after study day 1 (diet-only arm) until 30 days after last dose of the study. Serious adverse event (SAE).
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=19 Participants
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
AEs assessed by investigator as related to study drug
|
36 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
AEs leading to dose reduction
|
9 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
AEs leading to dose interruption
|
19 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
AEs leading to study discontinuation
|
1 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
Participants with Any SAE
|
2 Participants
|
1 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
SAEs assessed by investigator as related to study drug
|
2 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
SAEs leading to dose reduction
|
0 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
SAEs leading to dose interruption
|
0 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
SAEs leading to study drug discontinuation
|
2 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
SAEs leading to study discontinuation
|
1 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
Participants who died
|
0 Participants
|
0 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
Participants with Any AE
|
36 Participants
|
17 Participants
|
|
Incidence of Treatment-emergent Adverse Events (Part 1)
AEs leading to study drug discontinuation
|
2 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Week 73Population: Safety popln consists of all participants(partp) who recd atleast 1dose of study treat(peg/diet-only mgmt of PKU).Subj will be analyzed according to trtmt actually recd for analysis of safety. Anti-drug antibody samples are only drawn if partp is on trtmt drug.Since partp in"Diet-Only"arm are not taking peg in Part1 of study, there is no data to report. Of 32 partp completed wk 73, 1partp stayed in study but discontinued study drug \>30days prior wk73, hence data was excld from summary
The anti-pegvaliase total antibody (TAb) method captures drug-binding antibodies comprised of different isotypes and specificities. Antibody Positivity = number of subjects testing positive at any study visit / total number of subjects at study visit. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage of Participants With Positive Anti-pegvaliase Total Antibody (TAb)
Week 73
|
100 percentage of participants
|
—
|
|
Percentage of Participants With Positive Anti-pegvaliase Total Antibody (TAb)
Day 1
|
100 percentage of participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Week 73Population: Safety popln consists of all participants(partp) who recd atleast 1dose of study treat(peg/diet-only mgmt of PKU).Subj will be analyzed according to trtmt actually recd for analysis of safety. Anti-drug antibody samples are only drawn if partp is on trtmt drug.Since partp in"Diet-Only"arm are not taking peg in Part1 of study, there is no data to report. Of 32 partp completed wk 73, 1partp stayed in study but discontinued study drug \>30days prior wk73, hence data was excld from summary
The presence of immunoglobulin G (IgG) Antibodies against phenylalanine ammonia lyase (PAL) is measured overtime. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage of Participants With Positive PAL IgG Antibody
Day 1
|
22.2 percentage of participants
|
—
|
|
Percentage of Participants With Positive PAL IgG Antibody
Week 73
|
100 percentage of participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Week 73Population: Safety popln consists of all participants(partp) who recd atleast 1dose of study treat(peg/diet-only mgmt of PKU).Subj will be analyzed according to trtmt actually recd for analysis of safety. Anti-drug antibody samples are only drawn if partp is on trtmt drug.Since partp in"Diet-Only"arm are not taking peg in Part1 of study, there is no data to report. Of 32 partp completed wk 73, 1partp stayed in study but discontinued study drug \>30days prior wk73, hence data was excld from summary
The presence of immunoglobulin M (IgM) Antibodies against PAL (phenylalanine ammonia lyase) is measured overtime. PAL IgM was positive if the titer value was greater than or equal to the median of all baseline PAL IgM titers. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage of Participants With Positive PAL IgM Antibody
Day 1
|
50.0 percentage of participants
|
—
|
|
Percentage of Participants With Positive PAL IgM Antibody
Week 73
|
74.2 percentage of participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Week 73Population: Safety popln consists of all participants(partp) who recd atleast 1dose of study treat(peg/diet-only mgmt of PKU).Subj will be analyzed according to trtmt actually recd for analysis of safety. Anti-drug antibody samples are only drawn if partp is on trtmt drug.Since partp in"Diet-Only"arm are not taking peg in Part1 of study, there is no data to report. Of 32 partp completed wk 73, 1partp stayed in study but discontinued study drug \>30days prior wk73, hence data was excld from summary
The presence of IgG Antibodies against polyethylene glycol (PEG) is measured overtime The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage of Participants With Positive PEG IgG Antibody
Day 1
|
55.6 percentage of participants
|
—
|
|
Percentage of Participants With Positive PEG IgG Antibody
Week 73
|
3.2 percentage of participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Week 73Population: Safety popln consists of all participants(partp) who recd atleast 1dose of study treat(peg/diet-only mgmt of PKU).Subj will be analyzed according to trtmt actually recd for analysis of safety. Anti-drug antibody samples are only drawn if partp is on trtmt drug.Since partp in"Diet-Only"arm are not taking peg in Part1 of study, there is no data to report. Of 32 partp completed wk 73, 1partp stayed in study but discontinued study drug \>30days prior wk73, hence data was excld from summary
The presence of IgM Antibodies against PEG (polyethylene glycol) is measured overtime The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage of Participants With Positive PEG IgM Antibody
Day 1
|
100 percentage of participants
|
—
|
|
Percentage of Participants With Positive PEG IgM Antibody
Week 73
|
100 percentage of participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1), Week 73Population: Saf popln consists of all partp who recd atleast 1dose of study treat(peg/diet-only mgmt of PKU).Subj will be analyzed according to trtmt actually recd for analysis of saf.Anti-drug antibody samples are only drawn if partp is on trtmt drug.Since partp in"Diet-Only"arm are not taking peg in Part1 of study,there is no data to report.Of 32 partp completed wk73,1partp stayed in study but discontinued study drug\>30D prior wk73,hence data was excld from summary\&1partp didnt have NAb collected at wk73
Neutralizing antibodies (NAbs) capable of inhibiting the enzymatic activity of pegvaliase were measured. The baseline was defined as the last non-missing measurement collected prior to the first pegvaliase dose (pegvaliase arm) or prior to or on Visit 1 date (diet-only arm)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=36 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage of Participants With Positive Neutralizing Antibodies (NAb)
Day 1
|
8.3 percentage of participants
|
—
|
|
Percentage of Participants With Positive Neutralizing Antibodies (NAb)
Week 73
|
96.7 percentage of participants
|
—
|
SECONDARY outcome
Timeframe: Baseline to Week 72 (average of Week 69 and week 73)Population: The Intent to Treat (ITT) population will consist of all participants who are randomized. Participants will be analyzed according to the randomized treatment for the analysis of efficacy. Pegvalaise arm: Of the 32 participants who completed week 73, 1partp stayed in study but discontinued study drug \>30days prior wk73, hence data was excluded from summary Diet only arm: Of the 19 participants, 17 subjects completed week 73.
The total dietary protein intake following 72 weeks on study is defined as the mean total dietary protein intake calculated from the 3-day diet diaries collected at Week 69 and Week 73 visits. Total dietary protein is measured in grams/kilogram (g/kg). Baseline total protein intake at Part 1 was defined as the mean total dietary protein intake calculated from the 3 separate 3-day diet diaries collected during Screening/Run-in (2) and Day 1 (1).
Outcome measures
| Measure |
Experimental: Pegvaliase
n=31 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=17 Participants
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Change From Baseline in Total Dietary Protein Intake (i.e., Medical Food and/or Intact Food) Following 72 Weeks (Average of Week 69 and Week 73) on Study
Total Protein Intake
|
0.00 g/kg
Standard Deviation 0.267
|
0.01 g/kg
Standard Deviation 0.226
|
|
Change From Baseline in Total Dietary Protein Intake (i.e., Medical Food and/or Intact Food) Following 72 Weeks (Average of Week 69 and Week 73) on Study
From Medical Food
|
-0.20 g/kg
Standard Deviation 0.341
|
0.03 g/kg
Standard Deviation 0.216
|
|
Change From Baseline in Total Dietary Protein Intake (i.e., Medical Food and/or Intact Food) Following 72 Weeks (Average of Week 69 and Week 73) on Study
From Intact Food
|
0.21 g/kg
Standard Deviation 0.369
|
-0.02 g/kg
Standard Deviation 0.066
|
SECONDARY outcome
Timeframe: Baseline to Week 72 (average of Week 69 and week 73)Population: The Intent to Treat (ITT) population will consist of all participants who are randomized. Participants will be analyzed according to the randomized treatment for the analysis of efficacy. Pegvalaise arm:Of 32 partp who completed week73, 1partp stayed in study but discontinued study drug\>30days prior wk73, hence data was excluded from summary.1 partp(from medical food category)with baseline value=0,so %change is undefined Diet only arm: Of the 19 participants, 17 subjects completed week 73.
The total dietary protein intake following 72 weeks on study is defined as the mean total dietary protein intake calculated from the 3-day diet diaries collected at Week 69 and Week 73 visits. Total dietary protein is measured in grams/kilogram (g/kg). Baseline total protein intake at Part 1 was defined as the mean total dietary protein intake calculated from the 3 separate 3-day diet diaries collected during Screening/Run-in (2) and Day 1 (1).
Outcome measures
| Measure |
Experimental: Pegvaliase
n=31 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=17 Participants
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Percentage Change From Baseline in Total Dietary Protein Intake (i.e., Medical Food and/or Intact Food) Following 72 Weeks (Average of Week 69 and Week 73) on Study
Total Protein Intake
|
6.66 percentage change
Standard Deviation 32.249
|
10.68 percentage change
Standard Deviation 43.144
|
|
Percentage Change From Baseline in Total Dietary Protein Intake (i.e., Medical Food and/or Intact Food) Following 72 Weeks (Average of Week 69 and Week 73) on Study
From Medical Food
|
-28.79 percentage change
Standard Deviation 42.498
|
21.56 percentage change
Standard Deviation 73.388
|
|
Percentage Change From Baseline in Total Dietary Protein Intake (i.e., Medical Food and/or Intact Food) Following 72 Weeks (Average of Week 69 and Week 73) on Study
From Intact Food
|
106.66 percentage change
Standard Deviation 176.008
|
-10.00 percentage change
Standard Deviation 22.828
|
SECONDARY outcome
Timeframe: Week 73Population: The PK analysis population includes all participants who received pegvaliase and had at least one Week 73 intensive PK concentration measurement. At Week 73, PK concentration data were available for 24 subjects and these subjects were included in the concentration-time summaries. However, for the PK parameter summaries, 23 subjects contributed evaluable data. One subject in the 60 mg cohort had Week 73 concentration data available but no evaluable PK parameters were derived.
Time to maximum observed plasma concentration (Tmax) for pegvaliase during the Week 73 intensive PK assessment, defined as the elapsed time from dosing to the sampling time at which the highest observed pegvaliase plasma concentration (Cmax) occurs based on the Week 73 plasma concentration-time profile. Pharmacokinetic (PK)
Outcome measures
| Measure |
Experimental: Pegvaliase
n=23 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Pegvaliase Tmax (Week 73 Intensive PK)
Pegvaliase 60 mg
|
8.66 hrs
Standard Deviation 6.04
|
—
|
|
Pegvaliase Tmax (Week 73 Intensive PK)
Pegvaliase 20 mg
|
12.0 hrs
Standard Deviation NA
As the number of subjects assessed in this category is only 1, standard deviation (SD) is not applicable. Hence SD value is proposed as NA.
|
—
|
|
Pegvaliase Tmax (Week 73 Intensive PK)
Pegvaliase 40 mg
|
10.7 hrs
Standard Deviation 5.08
|
—
|
SECONDARY outcome
Timeframe: Week 73Population: The PK analysis population includes all participants who received pegvaliase and had at least one Week 73 intensive PK concentration measurement. At Week 73, PK concentration data were available for 24 subjects and these subjects were included in the concentration-time summaries. However, for the PK parameter summaries, 23 subjects contributed evaluable data. One subject in the 60 mg cohort had Week 73 concentration data available but no evaluable PK parameters were derived.
Maximum observed plasma concentration (Cmax) of pegvaliase during the Week 73 intensive PK assessment, defined as the highest measured pegvaliase plasma concentration across the Week 73 postdose sampling interval. Cmax is summarized in ng/mL concentration units based on observed values from the Week 73 concentration-time profile.
Outcome measures
| Measure |
Experimental: Pegvaliase
n=23 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Pegvaliase Cmax (Week 73 Intensive PK)
Pegvaliase 20 mg
|
2330 ng/mL
Standard Deviation NA
As the number of subjects assessed in this category is only 1, standard deviation (SD) is not applicable. Hence SD value is proposed as NA.
|
—
|
|
Pegvaliase Cmax (Week 73 Intensive PK)
Pegvaliase 40 mg
|
2480 ng/mL
Standard Deviation 3810
|
—
|
|
Pegvaliase Cmax (Week 73 Intensive PK)
Pegvaliase 60 mg
|
1750 ng/mL
Standard Deviation 2200
|
—
|
SECONDARY outcome
Timeframe: Week 73Population: The PK analysis population includes all participants who received pegvaliase and had at least one Week 73 intensive PK concentration measurement. At Week 73, PK concentration data were available for 24 subjects and these subjects were included in the concentration-time summaries. However, for the PK parameter summaries, 11 subjects contributed evaluable data. 13 subjects had Week 73 concentration data available but no evaluable PK parameters were derived.
Area under the plasma concentration-time curve from 0-24 hours postdose (AUC0-24) for pegvaliase during the Week 73 intensive PK assessment. AUC0-24 was calculated form the Week 73 plasma concentration-time data using noncompartmental methods and reported in ng\*h/mL units.
Outcome measures
| Measure |
Experimental: Pegvaliase
n=11 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Pegvaliase AUC0-24 (Week 73 Intensive PK)
Pegvaliase 20 mg
|
46600 h*ng/mL
Standard Deviation NA
As the number of subjects assessed in this category is only 1, Standard Deviation (SD) is not applicable. Hence SD values are proposed as NA.
|
—
|
|
Pegvaliase AUC0-24 (Week 73 Intensive PK)
Pegvaliase 40 mg
|
17000 h*ng/mL
Standard Deviation 27500
|
—
|
|
Pegvaliase AUC0-24 (Week 73 Intensive PK)
Pegvaliase 60 mg
|
23200 h*ng/mL
Standard Deviation 34000
|
—
|
SECONDARY outcome
Timeframe: Week 73Population: The PK analysis population includes all participants who received pegvaliase and had at least one Week 73 intensive PK concentration measurement. At Week 73, PK concentration data were available for 24 subjects and these subjects were included in the concentration-time summaries. However, for the PK parameter summaries, 15 subjects contributed evaluable data. 9 subjects had Week 73 concentration data available but no evaluable PK parameters were derived.
Average plasma concentration over the 0 to 24 hour postdose interval for pegvaliase during the Week 73 intensive PK assessment, reported in ng/mL concentration units, representing the mean pegvaliase exposure across the 24 hour postdose interval at Week 73.
Outcome measures
| Measure |
Experimental: Pegvaliase
n=15 Participants
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Pegvaliase Cavg (Week 73 Intensive PK)
Pegvaliase 20 mg
|
1940 ng/mL
Standard Deviation NA
As the number of subjects assessed in this category is only 1, standard deviation (SD) is not applicable. Hence SD value is proposed as NA
|
—
|
|
Pegvaliase Cavg (Week 73 Intensive PK)
Pegvaliase 40 mg
|
2740 ng/mL
Standard Deviation 4300
|
—
|
|
Pegvaliase Cavg (Week 73 Intensive PK)
Pegvaliase 60 mg
|
1550 ng/mL
Standard Deviation 2070
|
—
|
SECONDARY outcome
Timeframe: Up to Week 153Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline (Day 1), Week 145Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 145Outcome measures
Outcome data not reported
Adverse Events
Experimental: Pegvaliase
Serious events: 2 serious events
Other events: 36 other events
Deaths: 0 deaths
Control: Diet Only
Serious events: 1 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Experimental: Pegvaliase
n=36 participants at risk
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=19 participants at risk
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
Immune system disorders
Anaphylactic reaction
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Congenital, familial and genetic disorders
Hyperphenylalaninaemia
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
Other adverse events
| Measure |
Experimental: Pegvaliase
n=36 participants at risk
Pegvaliase: Induction Dose of 2.5 mg once weekly (QW), Titration dose of 2.5 mg twice weekly (BIW) up to 10 mg/kg once daily (QD), Maintenance dose of 20 mg QD up to 60 mg QD, administered subcutaneous (SC) using a prefilled syringe (PFS).
|
Control: Diet Only
n=19 participants at risk
Participants will be managing their PKU with diet alone. Participants in the diet-only control arm will be required to maintain and adjust dietary and medical protein food intake through Week 72.
|
|---|---|---|
|
General disorders
Injection site erythema
|
77.8%
28/36 • Number of events 269 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site pain
|
61.1%
22/36 • Number of events 65 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site pruritus
|
52.8%
19/36 • Number of events 151 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site swelling
|
47.2%
17/36 • Number of events 135 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Pyrexia
|
41.7%
15/36 • Number of events 20 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site induration
|
33.3%
12/36 • Number of events 115 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Fatigue
|
22.2%
8/36 • Number of events 13 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site bruising
|
22.2%
8/36 • Number of events 39 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site oedema
|
16.7%
6/36 • Number of events 58 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site warmth
|
19.4%
7/36 • Number of events 48 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site rash
|
16.7%
6/36 • Number of events 10 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site urticaria
|
13.9%
5/36 • Number of events 61 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Malaise
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site inflammation
|
5.6%
2/36 • Number of events 7 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Pain
|
5.6%
2/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Influenza like illness
|
5.6%
2/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site discolouration
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Peripheral swelling
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Feeling hot
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
General disorders
Injection site reaction
|
16.7%
6/36 • Number of events 28 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
75.0%
27/36 • Number of events 106 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
27.8%
10/36 • Number of events 16 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
13.9%
5/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
19.4%
7/36 • Number of events 11 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
6/36 • Number of events 8 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
15.8%
3/19 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
5.6%
2/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Rhabdomyolysis
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Nervous system disorders
Headache
|
66.7%
24/36 • Number of events 73 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
42.1%
8/19 • Number of events 12 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Nervous system disorders
Dizziness
|
30.6%
11/36 • Number of events 24 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Nervous system disorders
Tremor
|
5.6%
2/36 • Number of events 6 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Nausea
|
30.6%
11/36 • Number of events 22 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
15.8%
3/19 • Number of events 6 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Vomiting
|
27.8%
10/36 • Number of events 19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Abdominal pain
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
11.1%
4/36 • Number of events 6 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
15.8%
3/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Dyspepsia
|
8.3%
3/36 • Number of events 7 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Angular cheilitis
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Aphthous ulcer
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Dental discomfort
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Gastric ulcer
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Gingival pruritus
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Tooth development disorder
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Blood creatine phosphokinase increased
|
11.1%
4/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Complement factor C3 decreased
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Complement factor C4 decreased
|
11.1%
4/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Alanine aminotransferase increased
|
5.6%
2/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Amino acid level decreased
|
25.0%
9/36 • Number of events 11 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Aspartate aminotransferase increased
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Blood glucose increased
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
C-reactive protein increased
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Crystal urine present
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Haematocrit decreased
|
5.6%
2/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Haemoglobin increased
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Lymphocyte count increased
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Neutrophil count decreased
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Serum ferritin decreased
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Urine oxalate increased
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Investigations
Urobilinogen urine increased
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Upper respiratory tract infection
|
41.7%
15/36 • Number of events 23 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
36.8%
7/19 • Number of events 8 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Influenza
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Pharyngitis
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Pharyngitis streptococcal
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Urinary tract infection
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Asymptomatic COVID-19
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Bronchitis
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
COVID-19
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
21.1%
4/19 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Conjunctivitis
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Gastroenteritis viral
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Nail infection
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
4/36 • Number of events 8 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
26.3%
5/19 • Number of events 6 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Otitis externa
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Otitis media
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Rhinitis
|
5.6%
2/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
22.2%
8/36 • Number of events 19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
36.8%
7/19 • Number of events 15 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
25.0%
9/36 • Number of events 11 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
36.8%
7/19 • Number of events 13 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
11.1%
4/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
22.2%
8/36 • Number of events 19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
31.6%
6/19 • Number of events 13 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
13.9%
5/36 • Number of events 7 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 6 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
16.7%
6/36 • Number of events 7 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Rash
|
19.4%
7/36 • Number of events 11 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
19.4%
7/36 • Number of events 45 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
13.9%
5/36 • Number of events 11 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
11.1%
4/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Skin induration
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Dermatitis acneiform
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
5.6%
2/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Blood and lymphatic system disorders
Eosinophilia
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Blood and lymphatic system disorders
Anaemia
|
11.1%
4/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
8.3%
3/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Blood and lymphatic system disorders
Leukopenia
|
2.8%
1/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Blood and lymphatic system disorders
Lymph node pain
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Psychiatric disorders
Anxiety
|
11.1%
4/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Psychiatric disorders
Fear of injection
|
11.1%
4/36 • Number of events 7 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Psychiatric disorders
Aggression
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Psychiatric disorders
Autism spectrum disorder
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Psychiatric disorders
Panic attack
|
11.1%
4/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Psychiatric disorders
Procedural anxiety
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Contusion
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Limb injury
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Burns first degree
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Sunburn
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
11.1%
4/36 • Number of events 5 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Immune system disorders
Anaphylactic reaction
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Immune system disorders
Allergy to arthropod bite
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Immune system disorders
Seasonal allergy
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Eye disorders
Eye pruritus
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Eye disorders
Ocular hyperaemia
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
8.3%
3/36 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Vascular disorders
Flushing
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Congenital, familial and genetic disorders
Hyperphenylalaninaemia
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Ear and labyrinth disorders
Middle ear inflammation
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Ear and labyrinth disorders
Vertigo
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Endocrine disorders
Goitre
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Pregnancy, puerperium and perinatal conditions
Complication of pregnancy
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Pregnancy, puerperium and perinatal conditions
Morning sickness
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Renal and urinary disorders
Dysuria
|
5.6%
2/36 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
0.00%
0/19 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Renal and urinary disorders
Orthostatic proteinuria
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Renal and urinary disorders
Proteinuria
|
8.3%
3/36 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 3 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Nervous system disorders
Paraesthesia
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
0.00%
0/36 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
10.5%
2/19 • Number of events 2 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
5.3%
1/19 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
|
Gastrointestinal disorders
Diarrhoea
|
2.8%
1/36 • Number of events 1 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
21.1%
4/19 • Number of events 4 • Up to Week 73 (Part 1)
The Safety population will consist of all participants who received at least one dose of study treatment (pegvaliase or diet-only management of PKU). Subjects will be analyzed according to the treatment actually received for the analysis of safety.
|
Additional Information
Thomas Machnig, Senior Medical Director
BioMarin Pharmaceutical Inc.
Phone: +44 207 4203405
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER