Trial Outcomes & Findings for Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects Under Fed Conditions (NCT NCT06987773)
NCT ID: NCT06987773
Last Updated: 2026-04-28
Results Overview
The area under the analyte concentration versus time curve from time zero to the time of the last measurable analyte concentration (t)
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
42 participants
Primary outcome timeframe
Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)
Results posted on
2026-04-28
Participant Flow
Participant milestones
| Measure |
Sequence AB
The Participant receives in this order A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water followed by B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water
RLF-OD032 100 mg/mL oral suspension: Sapropterin dihydrochloride
Kuvan 100 MG Powder for Oral Solution: Sapropterin dihydrochloride
|
Sequence BA
The Participant receives in this order B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water followed by A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032), administered under fed conditions without water
RLF-OD032 100 mg/mL oral suspension: Sapropterin dihydrochloride
Kuvan 100 MG Powder for Oral Solution: Sapropterin dihydrochloride
|
|---|---|---|
|
Overall Study
STARTED
|
21
|
21
|
|
Overall Study
COMPLETED
|
20
|
20
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Sequence AB
The Participant receives in this order A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water followed by B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water
RLF-OD032 100 mg/mL oral suspension: Sapropterin dihydrochloride
Kuvan 100 MG Powder for Oral Solution: Sapropterin dihydrochloride
|
Sequence BA
The Participant receives in this order B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water followed by A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032), administered under fed conditions without water
RLF-OD032 100 mg/mL oral suspension: Sapropterin dihydrochloride
Kuvan 100 MG Powder for Oral Solution: Sapropterin dihydrochloride
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Non Compliance
|
0
|
1
|
Baseline Characteristics
Study on Sapropterin Dihydrochloride Oral Suspension in Healthy Subjects Under Fed Conditions
Baseline characteristics by cohort
| Measure |
All Patients Included in the 2 Sequences (AB,BA)
n=42 Participants
Where the Subject receives these treatments according to the order of the assigned sequence A) A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032), administered under fed conditions without water B) A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water followed by
RLF-OD032 100 mg/mL oral suspension: Sapropterin dihydrochloride
Kuvan 100 MG Powder for Oral Solution: Sapropterin dihydrochloride
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
42 Participants
n=9 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=9 Participants
|
|
Age, Continuous
|
36 years
STANDARD_DEVIATION 9 • n=9 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
|
8 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Black or African American
|
16 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Region of Enrollment
Canada
|
42 participants
n=9 Participants
|
PRIMARY outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Population: One (1) subject exhibited a pre-dose concentration for uncorrected sapropterin greater than 5% of their corresponding Cmax (9.17%) in Period 1 (Treatment A) and was excluded from the statistical dataset.
The area under the analyte concentration versus time curve from time zero to the time of the last measurable analyte concentration (t)
Outcome measures
| Measure |
Treatment A
n=40 Participants
Where A is A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water
|
Treatment B
n=40 Participants
Where B is A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water
|
|---|---|---|
|
Uncorrected and Baseline-corrected Sapropterin AUCt
Uncorrected Sapropterin
|
819.78 hr*ng/mL
Standard Error 32
|
805.79 hr*ng/mL
Standard Error 29
|
|
Uncorrected and Baseline-corrected Sapropterin AUCt
baseline-corrected Sapropterin
|
757.73 hr*ng/mL
Standard Error 34
|
755.15 hr*ng/mL
Standard Error 29
|
PRIMARY outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Population: AUCinf could not be estimated for one (1) subject following the administration of Treatment A due to poor goodness of fit in the terminal elimination phase and One (1) subject exhibited a pre-dose concentration for uncorrected sapropterin greater than 5% of their corresponding Cmax (9.17%) in Period 1 (Treatment A) and was excluded from the statistical dataset.
The area under the analyte concentration versus time curve from time zero to infinity
Outcome measures
| Measure |
Treatment A
n=40 Participants
Where A is A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water
|
Treatment B
n=40 Participants
Where B is A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water
|
|---|---|---|
|
Uncorrected and Baseline-corrected Sapropterin AUCinf
Uncorrected Sapropterin
|
868.74 hr*ng/mL
Standard Error 31
|
850.63 hr*ng/mL
Standard Error 28
|
|
Uncorrected and Baseline-corrected Sapropterin AUCinf
Baseline-corrected Sapropterin
|
777.88 hr*ng/mL
Standard Error 33
|
769.56 hr*ng/mL
Standard Error 28
|
PRIMARY outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Population: One (1) subject exhibited a pre-dose concentration for uncorrected sapropterin greater than 5% of their corresponding Cmax (9.17%) in Period 1 (Treatment A) and was excluded from the statistical dataset.
Maximum measured plasma analyte concentration over the sampling period
Outcome measures
| Measure |
Treatment A
n=40 Participants
Where A is A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water
|
Treatment B
n=40 Participants
Where B is A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water
|
|---|---|---|
|
Uncorrected and Baseline-corrected Sapropterin Cmax
Baseline-corrected Sapropterin
|
114.84 ng/mL
Standard Error 40
|
121.97 ng/mL
Standard Error 30
|
|
Uncorrected and Baseline-corrected Sapropterin Cmax
Uncorrected Sapropterin
|
119.25 ng/mL
Standard Error 42
|
124.09 ng/mL
Standard Error 30
|
SECONDARY outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Time of the maximum measured analyte concentration over the sampling period
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Time of observation prior to the first observation with a measurable (non-zero) concentration
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)The apparent elimination half-life
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Percentage of AUC0-inf due to extrapolation from the time of the last observed concentration to infinity, calculated as \[1 - (AUC0-t/AUC0-inf)\] x 100
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Apparent first-order elimination rate constant
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Apparent Volume of Distribution
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Pre-dose (-1, -0.5, and 0-hour) and at 0.333, 0.667, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 8, 10, 12, 16, 20, and 24 hours post-dose (22 time points)Apparent clearance
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Safety monitored from the signature of the Informed Consent through study completion (approximately 10 days)AEs will be coded using the latest version of the Medical Dictionary for Regulatory Activities (MedDRA).
Outcome measures
Outcome data not reported
Adverse Events
Treatment A
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Treatment B
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment A
n=41 participants at risk
Where A is A single 10 mg/kg dose of a 100 mg/mL oral suspension of sapropterin dihydrochloride (RLF-OD032) , administered under fed conditions without water
|
Treatment B
n=41 participants at risk
Where B is A single 10 mg/kg dose of a 100 mg powder for oral solution of the Reference Product (Kuvan) , dissolved in water, administered under fed conditions with water
|
|---|---|---|
|
Nervous system disorders
Somnolence
|
2.4%
1/41 • Number of events 1 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
0.00%
0/41 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/41 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
2.4%
1/41 • Number of events 1 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/41 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
2.4%
1/41 • Number of events 1 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
|
Investigations
White blood cell count decreased
|
0.00%
0/41 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
2.4%
1/41 • Number of events 1 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
|
Nervous system disorders
Headache
|
0.00%
0/41 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
2.4%
1/41 • Number of events 1 • 4 weeks (From ICF signature to the last contact to collect any safety follow-up data.
|
Additional Information
Giorgio Reiner Chief Scientific Officer
APR Applied Pharma Research s.a.
Phone: +41.91.6957020
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee There is an agreement between the Principal Investigator and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
- Publication restrictions are in place
Restriction type: OTHER