Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03392974 · ClinicalTrials.gov registry record · Phase 3
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
A Phase 3 study, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 1
- Enrollment target
NCT03392974: Completed Phase 3 study, sponsored by BioMarin Pharmaceutical.
NCT03392974 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 1 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (100% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03392974, a Phase 3 study, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 1 participants
- Enrollment target
Study Summary
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
Primary Outcome
Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.
Interventions
- BIOLOGICAL Valoctocogene Roxaparvovec
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 1 participants |
| Start Date | 2018-03-14 |
| Est. Completion | 2023-06-05 |
| Phase | Phase 3 |
What the finished NCT03392974 record still lists
NCT03392974 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 1 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (100% lower).
The record links to 0 conditions, and to 1 intervention - of which Valoctocogene Roxaparvovec is the first listed.
NCT03392974 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03392974 about?
NCT03392974 is a clinical study titled "Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg". This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
What is the current status of trial NCT03392974?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 1 participants. The study started on 2018-03-14. Estimated completion is 2023-06-05.
What interventions are being tested in trial NCT03392974?
The interventions under investigation include: Valoctocogene Roxaparvovec (BIOLOGICAL).
Who is sponsoring clinical trial NCT03392974?
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 NCT03392974's enrollment target sits among peer trials
1 2001st of 2000 the lowest 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI