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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 is a Phase 2 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 32 participants.

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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.

Interventions

  • DRUG Sapropterin Dihydrochloride

Trial Details

FieldValue
Enrollment Target 32 participants
Start Date 2007-05
Est. Completion 2009-06
Phase Phase 2

Sponsor

BioMarin Pharmaceutical

80 total trials

What the Registry Record Tells You About NCT00445978

The ClinicalTrials.gov registry entry for NCT00445978 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.

The record links to 0 conditions, and to 1 intervention - of which Sapropterin Dihydrochloride is the first listed.

NCT00445978 reports 0 study locations.

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.