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NCT00838435 · ClinicalTrials.gov registry record · Phase 3
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
A Phase 3 study of Phenylketonuria, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 95
- Enrollment target
- 19
- Study locations
NCT00838435: Completed Phase 3 study of Phenylketonuria, sponsored by BioMarin Pharmaceutical.
NCT00838435 is a Phase 3 study of Phenylketonuria that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 95 participants, roughly in line with the 91-participant average among 12 other Phenylketonuria trials with a reported enrollment target. The trial reports 19 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00838435, a Phase 3 study of Phenylketonuria, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 95 participants
- Enrollment target
- 19
- Study locations
Study Summary
This multicenter, open label study is designed to evaluate the safety of Kuvan® and its effect on neurocognitive function, blood Phe concentration, and growth in children with PKU who are 0-6 years old.
Primary Outcome
Full Scale Intelligence Quotient (FSIQ) is a score derived through administration of selected subtests from age appropriate Wechsler Intelligence assessments. Weschler Preschool and Primary Scale of Intelligence (WPPSI)-III is used for children \>30 months and ≤6 years; and Weschler Intelligence Scale for Children (WISC)-IV is used for children \>6 years old. The outcome variable will be the FSIQ score from WPPSI-III and/or WISC-IV tests. FSIQ results can range from 40 being the lowest and 160
Conditions Studied
Interventions
- DRUG sapropterin dihydrochloride
Study Locations (19)
California
- - La Jolla
- - Orange
Ohio
- - Cleveland
- - Columbus
Ontario
- - Hamilton
- - Toronto
Quebec
- - Montreal
- - Sainte-Foy
Florida
- - Tampa
Illinois
- - Chicago
Massachusetts
- - Boston
Missouri
- - Kansas City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 95 participants |
| Start Date | 2009-02 |
| Est. Completion | 2018-09-14 |
| Phase | Phase 3 |
What the finished NCT00838435 record still lists
NCT00838435 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 95 participants, a relatively small participant target, roughly in line with the 91-participant average among 12 other Phenylketonuria trials with a reported enrollment target.
The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 1 intervention - of which sapropterin dihydrochloride is the first listed.
NCT00838435 lists 19 locations in 15 states (California, Ohio, Ontario).
Frequently Asked Questions
What is clinical trial NCT00838435 about?
NCT00838435 is a clinical study titled "Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU". This multicenter, open label study is designed to evaluate the safety of Kuvan® and its effect on neurocognitive function, blood Phe concentration, and growth in children with PKU who are 0-6 years old.
What is the current status of trial NCT00838435?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 95 participants. The study started on 2009-02. Estimated completion is 2018-09-14.
What conditions does trial NCT00838435 study?
This clinical trial studies the following conditions: Phenylketonuria.
What interventions are being tested in trial NCT00838435?
The interventions under investigation include: sapropterin dihydrochloride (DRUG).
Who is sponsoring clinical trial NCT00838435?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00838435 being conducted?
This trial has 19 study locations across California, Florida, Illinois, Massachusetts, Missouri. 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 NCT00838435's enrollment target sits among peer trials
95 6th of 12 higher than 7 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.
Source ClinicalTrials.gov registry export · 2026-08-08
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