A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
Public ClinicalTrials.gov record NCT00445978. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2a, Multicenter, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, and Efficacy of 6R-BH4 in Subjects With Sickle Cell Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00445978
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 32 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 15 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 2007 (Actual)
- Primary completion
- Aug 2008 (Actual)
- Completion
- Jun 2009 (Actual)
- Last update posted
- Feb 25, 2021
2007 – 2009
United States locations
- U.S. sites
- 12
- U.S. states
- 9
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Not listed | Washington D.C. | District of Columbia | 20060 | — |
| Not listed | Augusta | Georgia | 30912 | — |
| Not listed | Savannah | Georgia | 31404 | — |
| Not listed | Indianapolis | Indiana | 46260 | — |
| Not listed | Detroit | Michigan | 48201 | — |
| Not listed | Flint | Michigan | 48503 | — |
| Not listed | Hackensack | New Jersey | 07601 | — |
| Not listed | Chapel Hill | North Carolina | 27599 | — |
| Not listed | Philadelphia | Pennsylvania | 19134 | — |
| Not listed | Galveston | Texas | 77555 | — |
| Not listed | Norfolk | Virginia | 23507 | — |
| Not listed | Richmond | Virginia | 23298 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00445978, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 25, 2021 · Synced Oct 19, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00445978 live on ClinicalTrials.gov.