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NCT01425528 · ClinicalTrials.gov registry record · Phase 1
Study of Kuvan Treatment in Adults With GTPCH Deficiency
A Phase 1 study, sponsored by University of Utah.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
NCT01425528: Completed Phase 1 study, sponsored by University of Utah.
NCT01425528 is a Phase 1 study that has completed, run by University of Utah. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01425528, a Phase 1 study, has completed, sponsored by University of Utah.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
Study Summary
The purpose of this study is to identify the dose of Kuvan needed to normalize the levels of tetrahydrobiopterin (BH4) in patients with a Guanosine Triphosphate Cyclohydrolase (GTPCH) deficiency and to asses the potential impact of oral Kuvan on mood and function in individuals with GTPCH deficiency who may be experiencing symptoms of anxiety, depression, fatigue, trouble concentrating, or memory loss. This will be a phase one study. The investigators will monitor patients over a period of three to six months while on the medication. Medication levels will be monitored by measuring the BH4 levels in cerebral spinal fluid (CSF). Patients will undergo a baseline lumbar puncture and two follow-up lumbar punctures for this purpose. The investigators will also monitor emotional function and motor function.
Primary Outcome
Identify dosing range of oral Kuvan® necessary and sufficient to normalize CSF BH4 levels in adults with GTPCH Deficiency.
Interventions
- DRUG Sapropterin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2011-08 |
| Est. Completion | 2013-08 |
| Phase | Phase 1 |
What the finished NCT01425528 record still lists
NCT01425528 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 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
The record links to 0 conditions, and to 1 intervention - of which Sapropterin is the first listed.
NCT01425528 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01425528 about?
NCT01425528 is a clinical study titled "Study of Kuvan Treatment in Adults With GTPCH Deficiency". The purpose of this study is to identify the dose of Kuvan needed to normalize the levels of tetrahydrobiopterin (BH4) in patients with a Guanosine Triphosphate Cyclohydrolase (GTPCH) deficiency and to asses the potential impact of oral Kuvan on mood and function in individuals with GTPCH deficiency...
What is the current status of trial NCT01425528?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2011-08. Estimated completion is 2013-08.
What interventions are being tested in trial NCT01425528?
The interventions under investigation include: Sapropterin (DRUG).
Who is sponsoring clinical trial NCT01425528?
This trial is sponsored by University of Utah, which has 818 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01425528's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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