Trial Outcomes & Findings for Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria (NCT NCT01977820)
NCT ID: NCT01977820
Last Updated: 2018-02-22
Results Overview
An AE was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A SAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
Screening up to 24 weeks + 4-week follow-up
Results posted on
2018-02-22
Participant Flow
First/Last subject (informed consent): 24 Feb 2014/12 Aug 2014. Study premature termination date: 07 Nov 2014; Subjects were randomized at 2 study centers.
A total of 10 subjects were screened and 7 subjects were included in the 2-Week response test period. Out of 7 subjects, only 2 subjects were responders and they were enrolled and randomized to receive study drug into the 24-Week study period.
Participant milestones
| Measure |
Sapropterin
Subject was administered with 20 milligram per kilogram (mg/kg) sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive sapropterin during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
Placebo
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive placebo tablets matching to sapropterin orally once daily during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Sapropterin
Subject was administered with 20 milligram per kilogram (mg/kg) sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive sapropterin during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
Placebo
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive placebo tablets matching to sapropterin orally once daily during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
|---|---|---|
|
Overall Study
Sponsor terminated the study
|
1
|
1
|
Baseline Characteristics
Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
Baseline characteristics by cohort
| Measure |
Sapropterin
n=1 Participants
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive sapropterin during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
Placebo
n=1 Participants
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive placebo tablets matching to sapropterin orally once daily during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
>=18 to 29 Years
|
1 Subjects
n=93 Participants
|
1 Subjects
n=4 Participants
|
2 Subjects
n=27 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
1 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: Screening up to 24 weeks + 4-week follow-upPopulation: The safety analysis population included all the randomized subjects who received at least one dose of study treatment.
An AE was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A SAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect.
Outcome measures
| Measure |
Sapropterin
n=1 Participants
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive sapropterin during the 24-week study period. The subject completed the study according to the study protocol until the study was terminated by the Sponsor.
|
Placebo
n=1 Participants
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive placebo tablets matching to sapropterin orally once daily during the 24-week study period. The subject completed the study according to the study protocol until the study was terminated by the Sponsor.
|
|---|---|---|
|
Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation
AEs
|
1 Subjects
|
0 Subjects
|
|
Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation
SAEs
|
0 Subjects
|
0 Subjects
|
|
Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation
AEs leading to death
|
0 Subjects
|
0 Subjects
|
|
Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation
AEs leading to discontinuation
|
0 Subjects
|
0 Subjects
|
Adverse Events
Sapropterin
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Sapropterin
n=1 participants at risk
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive sapropterin during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
Placebo
n=1 participants at risk
Subject was administered with 20 mg/kg sapropterin tablets orally once daily during the 2-week response test period. The subject upon completing the 2-Week response test period was randomized to receive placebo tablets matching to sapropterin orally once daily during the 24-week study period. The subject underwent the study assessments and procedures according to the study protocol until the study was terminated by the Sponsor.
|
|---|---|---|
|
Nervous system disorders
Headache
|
100.0%
1/1 • Screening up to 24 weeks + 4-week follow-up
|
0.00%
0/1 • Screening up to 24 weeks + 4-week follow-up
|
|
Gastrointestinal disorders
Abdominal Pain
|
100.0%
1/1 • Screening up to 24 weeks + 4-week follow-up
|
0.00%
0/1 • Screening up to 24 weeks + 4-week follow-up
|
|
Gastrointestinal disorders
Aphthous stomatitis
|
100.0%
1/1 • Screening up to 24 weeks + 4-week follow-up
|
0.00%
0/1 • Screening up to 24 weeks + 4-week follow-up
|
|
Gastrointestinal disorders
Abdominal pain upper
|
100.0%
1/1 • Screening up to 24 weeks + 4-week follow-up
|
0.00%
0/1 • Screening up to 24 weeks + 4-week follow-up
|
Additional Information
Medical Information Services
BioMarin Pharmaceutical Inc.
Phone: +1-800-983-4587
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee All written or oral publications and/or information related to Study and/or results of Study should be submitted at first in writing to Sponsor at least 30 working days for publication and 15 working days for brief review, abstract before planned date of submission. If Sponsor is filing a patent application on Study results, Sponsor can delay its authorization for publication/communication of Study results to Investigator and/or Research Centre until date of international registration of patent.
- Publication restrictions are in place
Restriction type: OTHER