Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
Public ClinicalTrials.gov record NCT03370913. 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 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03370913
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 144 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- Male
- 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
- Dec 18, 2017
- Primary completion
- Nov 15, 2020
- Completion
- Nov 19, 2024
- Last update posted
- Mar 24, 2025
2017 – 2024
United States locations
- U.S. sites
- 15
- U.S. states
- 11
- U.S. cities
- 15
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center | Los Angeles | California | 90007-2664 | — |
| UC Davis Hemophilia Treatment Center | Sacramento | California | 95817 | — |
| University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center | San Diego | California | 92122 | — |
| UCSF Medical Center | San Francisco | California | 94143-0106 | — |
| University of Colorado | Aurora | Colorado | 80045 | — |
| St. Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders | Tampa | Florida | 33607 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago, Department of Hematology | Chicago | Illinois | 60611-2605 | — |
| James Graham Brown Cancer Center | Louisville | Kentucky | 40202 | — |
| Tulane University Hematology & Medical Oncology | New Orleans | Louisiana | 70112-2699 | — |
| University of Michigan, Pediatric Hematology and Oncology | Ann Arbor | Michigan | 48109-5718 | — |
| Wayne State University, Detroit Medical Center | Detroit | Michigan | 48201 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology | St Louis | Missouri | 63110-1093 | — |
| UNC Hemophilia and Thrombosis Center | Chapel Hill | North Carolina | 27517 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 34 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03370913, 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 Mar 24, 2025 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03370913 live on ClinicalTrials.gov.