# Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

> **NCT03520712** · PHASE1,PHASE2 · TERMINATED · sponsor: **BioMarin Pharmaceutical** · enrollment: 3 (actual)

## Conditions studied

- Hemophilia A
- Gene Therapy
- Clotting Disorders
- Blood Disorder

## Interventions

- **BIOLOGICAL:** Valoctocogene Roxaparvovec

## Key facts

- **NCT ID:** NCT03520712
- **Lead sponsor:** BioMarin Pharmaceutical
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2018-04-24
- **Primary completion:** 2024-08-07
- **Final completion:** 2024-08-07
- **Target enrollment:** 3 (ACTUAL)
- **Why stopped:** Early rollover into long-term extension study
- **Last updated:** 2025-08-22


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03520712

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03520712, "Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5". Retrieved via AI Analytics 2026-07-13 from https://api.ai-analytics.org/clinical/NCT03520712. Licensed CC0.

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