Trial Outcomes & Findings for Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU (NCT NCT03694353)
NCT ID: NCT03694353
Last Updated: 2022-02-17
Results Overview
A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
37 participants
Primary outcome timeframe
Up to Day 741 (approximately Week 106)
Results posted on
2022-02-17
Participant Flow
This was a multicenter study, conducted in 17 study centers in the United States involving subjects from either Parent Study 165-302 (NCT02468570) or Parent Study PAL-003 (NCT00924703).
Participant milestones
| Measure |
All Subjects
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
Overall Study
STARTED
|
37
|
|
Overall Study
COMPLETED
|
35
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
All Subjects
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Subject received <= 40 mg/day for > 32 weeks
|
1
|
Baseline Characteristics
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
Baseline characteristics by cohort
| Measure |
All Subjects
n=37 Participants
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
Age, Continuous
|
28.84 years
STANDARD_DEVIATION 9.02 • n=99 Participants
|
|
Age, Customized
16 to < 18 years
|
2 Participants
n=99 Participants
|
|
Age, Customized
Between 18 and < 66 years
|
35 Participants
n=99 Participants
|
|
Age, Customized
>/= 66 years
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
15 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
36 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
36 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Weight
|
94.9 kg
STANDARD_DEVIATION 29.75 • n=99 Participants
|
|
Body Mass Index (BMI)
|
32 kg/m^2
STANDARD_DEVIATION 8.41 • n=99 Participants
|
|
Baseline Blood Phe
|
1376.9 umol/L
STANDARD_DEVIATION 289.77 • n=99 Participants
|
PRIMARY outcome
Timeframe: Up to Day 741 (approximately Week 106)Population: Population consisted of all subjects receiving at least one dose of pegvaliase during the study.
A treatment-emergent AE was defined as any adverse event (AE) newly appearing or worsened in severity following initiation of study drug until 4 weeks after last dose of pegvaliase
Outcome measures
| Measure |
All Subjects
n=37 Participants
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
TEAE
|
36 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
Related TEAE
|
17 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
Serious TEAE
|
7 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) as Assessed by CTCAE v5.0
Related Serious TEAE
|
1 Participants
|
PRIMARY outcome
Timeframe: The Outcome Measure Data Table below uses the 'analysis visit' as defined by the mapping rule in the SAP. The last Phe measurement was mapped to 'analysis visit' week 121. The actual date of the last Phe measurement was day 836 (approximately Week 119).Population: Efficacy population consisted of all subjects receiving at least one dose of pegvaliase during the study and having a post-treatment blood Phe concentration measurement.
Change in blood phenylalanine (Phe) concentration from Parent Study baseline (naïve/pretreatment).
Outcome measures
| Measure |
All Subjects
n=37 Participants
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
Change in Blood Phe Concentration
Change from Baseline at Week 121
|
-1123 µmol/L
Standard Deviation NA
Only one subject at Week 121 therefore standard deviation is not applicable.
|
|
Change in Blood Phe Concentration
Parent Study Baseline (naïve/pretreatment)
|
1376.9 µmol/L
Standard Deviation 289.77
|
|
Change in Blood Phe Concentration
Change from Baseline to Screening/Day 1
|
-761 µmol/L
Standard Deviation 506.7
|
|
Change in Blood Phe Concentration
Change from Baseline at week 9
|
-970.6 µmol/L
Standard Deviation 513.6
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 17
|
-963.4 µmol/L
Standard Deviation 454
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 25
|
-830.6 µmol/L
Standard Deviation 599.8
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 33
|
-999.8 µmol/L
Standard Deviation 528
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 41
|
-984.1 µmol/L
Standard Deviation 545.6
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 49
|
-1001.7 µmol/L
Standard Deviation 518.3
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 57
|
-867 µmol/L
Standard Deviation 569.2
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 65
|
-913.2 µmol/L
Standard Deviation 445
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 73
|
-896 µmol/L
Standard Deviation 582.8
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 81
|
-847.8 µmol/L
Standard Deviation 504
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 89
|
-962.3 µmol/L
Standard Deviation 482.6
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 97
|
-865.6 µmol/L
Standard Deviation 567.4
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 105
|
-817.4 µmol/L
Standard Deviation 584.9
|
|
Change in Blood Phe Concentration
Change from Baseline at Week 113
|
-1319 µmol/L
Standard Deviation 123
|
Adverse Events
All Subjects
Serious events: 7 serious events
Other events: 36 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Subjects
n=37 participants at risk
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
Infections and infestations
COVID-19
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Wound infection staphylococcal
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Pregnancy, puerperium and perinatal conditions
Ectopic pregnancy
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Gastric ulcer haemorrhage
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Systemic inflammatory response syndrome
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Hepatobiliary disorders
Cholecystitis
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Immune system disorders
Anaphylactic reaction
|
2.7%
1/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
Other adverse events
| Measure |
All Subjects
n=37 participants at risk
Self administered subcutaneous pegvaliase injection using a prefilled syringe.
|
|---|---|
|
General disorders
Pyrexia
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Nasopharyngitis
|
35.1%
13/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Influenza
|
18.9%
7/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Gastroenteritis
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Upper respiratory tract infection
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Helicobacter infection
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Sinusitis
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Bronchitis
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Cellulitis
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Conjunctivitis
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Ear infection
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Gastroenteritis viral
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Otitis media acute
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Pharyngitis streptococcal
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Pneumonia
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Tinea pedis
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Infections and infestations
Tooth abscess
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Nausea
|
16.2%
6/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Vomiting
|
16.2%
6/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Diarrhoea
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Toothache
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Dental caries
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Injection site bruising
|
16.2%
6/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Fatigue
|
13.5%
5/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Injection site induration
|
13.5%
5/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Injection site reaction
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Asthenia
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Induration
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Injection site erythema
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Injection site pain
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Injection site pruritus
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
General disorders
Pain
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Nervous system disorders
Headache
|
32.4%
12/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Nervous system disorders
Migraine
|
13.5%
5/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Nervous system disorders
Dizziness
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Nervous system disorders
Hypoaesthesia
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Nervous system disorders
Paraesthesia
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Nervous system disorders
Sinus headache
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.2%
6/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
13.5%
5/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
16.2%
6/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
18.9%
7/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.2%
6/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal sinus discomfort
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Injury, poisoning and procedural complications
Limb injury
|
13.5%
5/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Injury, poisoning and procedural complications
Contusion
|
10.8%
4/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Investigations
Amino acid level decreased
|
18.9%
7/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Psychiatric disorders
Depression
|
8.1%
3/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Psychiatric disorders
Anxiety
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Immune system disorders
Seasonal allergy
|
13.5%
5/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
|
Vascular disorders
Hypertension
|
5.4%
2/37 • Up to Day 741 (approximately Week 106)
Only treatment-emergent AEs that occurred and were reported during the study period were included in AE summaries. The incidence, exposure-adjusted event rate, severity grade (assessed per CTCAE version 5.0), and relationship to study drug (per Investigator) for all treatment-emergent AEs were summarized. For AEs occurring more than once during the study, maximum severity was used to summarize AEs by severity.
|
Additional Information
Debra Lounsbury/Sr. Director, Clinical Sciences
BioMarin Pharmaceutical Inc
Phone: 415-506-6348
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60