Clinical trial
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
NCT02576795 · BioMarin Pharmaceutical, Industry · Completed
Key facts
| NCT number | NCT02576795 |
|---|---|
| Title | Gene Therapy Study in Severe Haemophilia A Patients (270-201) |
| Official title | A Phase 1/2, Dose-Escalation, Safety, Tolerability and Efficacy Study of Valoctocogene Roxaparvovec, an Adenovirus-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A |
| Lead sponsor | BioMarin Pharmaceutical, Industry |
| Status | Completed |
| Phase | Phase 1/Phase 2 |
| Study type | Interventional |
| Conditions | Severe Haemophilia A |
| Intervention type | Biological |
| Interventions | valoctocogene roxaparvovec |
| Enrollment | 15 |
| Start date | Sep 28, 2015 |
| Primary completion | Feb 14, 2024 |
| Completion | Feb 14, 2024 |
| First posted | Oct 15, 2015 |
| Last update posted | Apr 10, 2025 |
| Countries | United Kingdom |
At a glance
- 1–9 Study sites
- Yes Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: