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NCT00445978 · ClinicalTrials.gov registry record · Phase 2
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
A Phase 2 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 32
- Enrollment target
NCT00445978: Completed Phase 2 study, sponsored by BioMarin Pharmaceutical.
NCT00445978 is a Phase 2 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 32 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (76% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00445978, a Phase 2 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 32 participants
- Enrollment target
Study Summary
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with sickle cell disease. During an optional extension phase, the study will assess the safety, tolerability, and efficacy of extended treatment with sapropterin dihydrochloride, for a total of up to 2 years; The extension phase of this study was terminated.
Primary Outcome
A treatment-emergent adverse events (TEAE) is any adverse events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration.
Interventions
- DRUG Sapropterin Dihydrochloride
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2007-05 |
| Est. Completion | 2009-06 |
| Phase | Phase 2 |
What the finished NCT00445978 record still lists
NCT00445978 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (76% lower).
The record links to 0 conditions, and to 1 intervention - of which Sapropterin Dihydrochloride is the first listed.
NCT00445978 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00445978 about?
NCT00445978 is a clinical study titled "A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease". This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with sickle cell disease. During an optional extension phase, the study wil...
What is the current status of trial NCT00445978?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 32 participants. The study started on 2007-05. Estimated completion is 2009-06.
What interventions are being tested in trial NCT00445978?
The interventions under investigation include: Sapropterin Dihydrochloride (DRUG).
Who is sponsoring clinical trial NCT00445978?
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 NCT00445978's enrollment target sits among peer trials
32 1563rd of 2000 higher than 423 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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