Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

ClinicalTrials.gov ID: NCT03392974

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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 at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/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 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.

Study identification

NCT ID: NCT03392974
Recruitment status: Completed
Study type: Interventional
Phase: Phase 3
Lead sponsor: BioMarin Pharmaceutical; Industry
Enrollment: 1 participant

Conditions and interventions

Conditions

Hemophilia A

Interventions

Valoctocogene Roxaparvovec Biological

Biological

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: Mar 14, 2018 (Actual)
Primary completion: May 22, 2019 (Actual)
Completion: Jun 5, 2023 (Actual)
Last update posted: Oct 3, 2023

2018 – 2023

United States locations

U.S. sites: 1
U.S. states: 1
U.S. cities: 1

Facility	City	State	ZIP	Site status
Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania	15213-4306	—

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 NCT03392974, 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 Oct 3, 2023 · Synced Nov 14, 2026