3 completed, 1 active not recruiting. 4 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 4 | Completed 3, Active not recruiting 1 |
Follow Valoctocogene Roxaparvovec: new trials by email Literature Review of Valoctocogene Roxaparvovec trials Research Report on Valoctocogene Roxaparvovec
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A Valoctocogene roxaparvovec - Hemophilia A | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 6 | 2023-12-25 | 2026-03-19 |
| Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A valoctocogene roxaparvovec - Hemophilia A | Phase 3 | Completed | BioMarin Pharmaceutical | 22 | 2020-12-08 | 2025-10-08 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg Valoctocogene Roxaparvovec - Hemophilia A | Phase 3 | Completed | BioMarin Pharmaceutical | 1 | 2018-03-14 | 2023-10-03 |
| Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) valoctocogene roxaparvovec - Hemophilia A | Phase 3 | Completed | BioMarin Pharmaceutical | 144 | 2017-12-19 | 2025-03-25 |
Blood Coagulation Disorders (4) · Blood Coagulation Disorders, Inherited (4) · Coagulation Protein Disorders (4) · Genetic Diseases, Inborn (4) · Hematologic Diseases (4) · Hemophilia A (4) · Hemorrhagic Disorders (4) · Hemostatic Disorders (4)
United States (3) · Australia (2) · Brazil (2) · Taiwan (2) · Belgium (1) · France (1) · Germany (1) · Israel (1) · Italy (1) · Japan (1) · South Africa (1) · South Korea (1) · Spain (1) · United Kingdom (1)
PMID 32915950, PMID 35294811, PMID 35879931, PMID 36812433, PMID 38616241, PMID 39024543, PMID 40986187
2017 (2)