5 completed, 1 active not recruiting, 1 terminated. 4 Phase 3, 3 Phase 1, 3 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 4 | Completed 3, Active not recruiting 1 |
| Phase 1 | 3 | Completed 2, Terminated 1 |
| Phase 2 | 3 | Completed 2, Terminated 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 |
| Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors Valoctocogene roxaparvovec - Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 10 | 2020-12-10 | 2026-07-10 |
| 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 |
| Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 Valoctocogene Roxaparvovec - Hemophilia A, Gene Therapy | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 3 | 2018-04-24 | 2025-08-22 |
| 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 |
| Gene Therapy Study in Severe Haemophilia A Patients (270-201) valoctocogene roxaparvovec - Severe Haemophilia A | Phase 1 / Phase 2 | Completed | BioMarin Pharmaceutical | 15 | 2015-09-28 | 2025-04-10 |
Blood Coagulation Disorders (7) · Blood Coagulation Disorders, Inherited (7) · Coagulation Protein Disorders (7) · Genetic Diseases, Inborn (7) · Hematologic Diseases (7) · Hemophilia A (7) · Hemorrhagic Disorders (7) · Hemostatic Disorders (7)
Taiwan (4) · United Kingdom (4) · United States (4) · Brazil (3) · South Korea (3) · Australia (2) · Israel (2) · South Africa (2) · Belgium (1) · France (1) · Germany (1) · Italy (1) · Japan (1) · Spain (1) · Turkey (Türkiye) (1)
PMID 32915950, PMID 35879931, PMID 40986187, PMID 29224506, PMID 31893514, PMID 34378280, PMID 35294811, PMID 35411075, PMID 36812433, PMID 38616241, PMID 38975624, PMID 39024543
2017 (3), 2014 (1), 2019 (1)