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NCT00104260 · ClinicalTrials.gov registry record · Phase 2
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
A Phase 2 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 2
- Development phase
- 700
- Enrollment target
NCT00104260: Completed Phase 2 study, sponsored by BioMarin Pharmaceutical.
NCT00104260 is a Phase 2 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 700 participants, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (426% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00104260, a Phase 2 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 700 participants
- Enrollment target
Study Summary
The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary objective of this study is to evaluate the safety of Phenoptin™ treatment in this subject population, and identify individuals in this subject population who respond to Phenoptin™ treatment with a reduction in blood Phe level.
Interventions
- DRUG sapropterin dihydrochloride
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 700 participants |
| Start Date | 2004-12 |
| Est. Completion | 2005-11 |
| Phase | Phase 2 |
What the finished NCT00104260 record still lists
NCT00104260 is an interventional study that assigns participants to a tested intervention. The registered 700 participants enrollment target is mid-sized for trials with a published cap, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (426% higher).
The record links to 0 conditions, and to 1 intervention - of which sapropterin dihydrochloride is the first listed.
NCT00104260 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00104260 about?
NCT00104260 is a clinical study titled "Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria". The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary objective of this study is to e...
What is the current status of trial NCT00104260?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 700 participants. The study started on 2004-12. Estimated completion is 2005-11.
What interventions are being tested in trial NCT00104260?
The interventions under investigation include: sapropterin dihydrochloride (DRUG).
Who is sponsoring clinical trial NCT00104260?
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 NCT00104260's enrollment target sits among peer trials
700 46th of 2000 higher than 1,955 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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