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NCT01114737 · ClinicalTrials.gov registry record · Phase 3
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
A Phase 3 study of Phenylketonuria, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 206
- Enrollment target
- 20
- Study locations
NCT01114737: Completed Phase 3 study of Phenylketonuria, sponsored by BioMarin Pharmaceutical.
NCT01114737 is a Phase 3 study of Phenylketonuria that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 206 participants, above the 82-participant average among 12 other Phenylketonuria trials with a reported enrollment target (151% higher). The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01114737, a Phase 3 study of Phenylketonuria, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 206 participants
- Enrollment target
- 20
- Study locations
Study Summary
This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.
Primary Outcome
Effects of 6R-BH4 on symptoms of ADHD in PKU subjects who had symptoms of ADHD at screening in the subjects that had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Sapropterin dihydrochloride
Study Locations (20)
California
- - La Jolla
- - Los Angeles
- - Palo Alto
- - San Francisco
New York
- - Albany
- - Buffalo
- - Rochester
Florida
- - Gainesville
- - Tampa
Colorado
- - Aurora
District of Columbia
- - Washington D.C.
Georgia
- - Atlanta
Illinois
- - Chicago
Indiana
- - Indianapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 206 participants |
| Start Date | 2010-08 |
| Est. Completion | 2013-03 |
| Phase | Phase 3 |
What the finished NCT01114737 record still lists
NCT01114737 is an interventional study that assigns participants to a tested intervention. The registered 206 participants enrollment target is mid-sized for trials with a published cap, above the 82-participant average among 12 other Phenylketonuria trials with a reported enrollment target (151% higher).
The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT01114737 names 20 study sites across 14 states, led by California, New York, Florida.
Frequently Asked Questions
What is clinical trial NCT01114737 about?
NCT01114737 is a clinical study titled "Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients". This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.
What is the current status of trial NCT01114737?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 206 participants. The study started on 2010-08. Estimated completion is 2013-03.
What conditions does trial NCT01114737 study?
This clinical trial studies the following conditions: Phenylketonuria.
What interventions are being tested in trial NCT01114737?
The interventions under investigation include: Placebo (DRUG), Sapropterin dihydrochloride (DRUG).
Who is sponsoring clinical trial NCT01114737?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01114737 being conducted?
This trial has 20 study locations across California, Colorado, District of Columbia, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Phenylketonuria
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01114737's enrollment target sits among peer trials
206 1st of 12 higher than 12 of 12 other Phenylketonuria trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phenylketonuria trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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