Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

ClinicalTrials.gov ID: NCT04684940

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Official title

A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.

Study identification

NCT ID: NCT04684940
Recruitment status: Completed
Study type: Interventional
Phase: Phase 1, Phase 2
Lead sponsor: BioMarin Pharmaceutical; Industry
Enrollment: 10 participants

Conditions and interventions

Conditions

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: Dec 10, 2020 (Actual)
Primary completion: Apr 20, 2026 (Actual)
Completion: Apr 20, 2026 (Actual)
Last update posted: Jul 10, 2026

2020 – 2026

United States locations

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

Facility	City	State	ZIP	Site status
Children's Hospital Los Angeles	Los Angeles	California	90027	—

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 8 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT04684940, 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 Jul 10, 2026 · Synced Nov 12, 2026