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NCT03370913 · ClinicalTrials.gov registry record · Phase 3
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 144
- Enrollment target
NCT03370913: Completed Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT03370913 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 144 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (81% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03370913, a Phase 3 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 144 participants
- Enrollment target
Study Summary
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP) (from Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII prophylaxis plus the washout period (3 days for products of standard half-life or plasma-derived and 5 days for products of extended half-life), whichever is later, to last visit by the data cut-off for the 2-year analysis, hereafter referred to as "Post FVIII Prophylaxis to Last Visit"). The study will also assess the impact of BMN 270 (compared to FVIII prophylaxis) on: the number of bleeding episodes requiring exogenous FVIII treatment in "Post FVIII Prophylaxis to Last Visit", FVIII activity as measured by chromogenic sustrate assay at Week 104 following intravenous infusion of BMN 270, usage of exogenous FVIII replacement therapy in "Post FVIII Prophylaxis to Last Visit", health-related quality of life patient-reported outcomes at week 104 following intravenous infusion of BMN 270. The study will also evaluate the safety of the BMN 270.
Primary Outcome
All bleeds comprises both treated and non-treated bleeds. In this definition, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded. All bleeds are any reported bleeding events regardless of the use of FVIII or other treatments. ABR for all bleeds= Number of bleeding episodes for all bleeds during the calculation period / total number of days during the calculation period \* 365.25. Baseline: prio
Interventions
- BIOLOGICAL valoctocogene roxaparvovec
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 144 participants |
| Start Date | 2017-12-19 |
| Est. Completion | 2024-11-20 |
| Phase | Phase 3 |
What the finished NCT03370913 record still lists
NCT03370913 is an interventional study that assigns participants to a tested intervention. The registered 144 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (81% lower).
The record links to 0 conditions, and to 1 intervention - of which valoctocogene roxaparvovec is the first listed.
NCT03370913 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03370913 about?
NCT03370913 is a clinical study titled "Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)". This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP) (from Week 5 post-BMN 270 infusion (Study Day 33) or the end of FVIII proph...
What is the current status of trial NCT03370913?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 144 participants. The study started on 2017-12-19. Estimated completion is 2024-11-20.
What interventions are being tested in trial NCT03370913?
The interventions under investigation include: valoctocogene roxaparvovec (BIOLOGICAL).
Who is sponsoring clinical trial NCT03370913?
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 NCT03370913's enrollment target sits among peer trials
144 1559th of 2000 higher than 442 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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