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NCT00355264 · ClinicalTrials.gov registry record · Phase 2
Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
A Phase 2 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 12
- Enrollment target
NCT00355264: Completed Phase 2 study, sponsored by BioMarin Pharmaceutical.
NCT00355264 is a Phase 2 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 12 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00355264, a Phase 2 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 12 participants
- Enrollment target
Study Summary
The purpose of this study is to evaluate the ability of Phenoptin to control blood phenylalanine levels in subjects who have hyperphenylalaninemia due to a primary BH4 deficiency and to evaluate the safety of Phenoptin in this population. Some subjects were receiving non-registered formulations of BH4 at enrollment and this treatment was suspended after Part 1 and within one day the subjects started Phenoptin at approximately the same dose.
Primary Outcome
Baseline blood phenylalanine(Phe) value is the latest measurement taken prior to initiation of Phenoptin treatment. The ideal range for blood Phe levels is approximately 120-360 µmol/L.
Interventions
- DRUG Phenoptin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2006-08 |
| Est. Completion | 2009-06 |
| Phase | Phase 2 |
What the finished NCT00355264 record still lists
NCT00355264 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (91% lower).
The record links to 0 conditions, and to 1 intervention - of which Phenoptin is the first listed.
NCT00355264 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00355264 about?
NCT00355264 is a clinical study titled "Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency". The purpose of this study is to evaluate the ability of Phenoptin to control blood phenylalanine levels in subjects who have hyperphenylalaninemia due to a primary BH4 deficiency and to evaluate the safety of Phenoptin in this population. Some subjects were receiving non-registered formulations of B...
What is the current status of trial NCT00355264?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 12 participants. The study started on 2006-08. Estimated completion is 2009-06.
What interventions are being tested in trial NCT00355264?
The interventions under investigation include: Phenoptin (DRUG).
Who is sponsoring clinical trial NCT00355264?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00355264's enrollment target sits among peer trials
12 1948th of 2000 higher than 34 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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