1 active not recruiting, 1 withdrawn. 2 Phase 3, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 2 | Active not recruiting 1, Withdrawn 1 |
| Phase 2 | 1 | Withdrawn 1 |
Follow Genetic Therapy: new trials by email Literature Review of Genetic Therapy trials Research Report on Genetic Therapy
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 |
|---|---|---|---|---|---|---|
| An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy - Hemophilia A | Phase 3 | Active not recruiting | Pfizer | 77 | 2020-08-18 | 2026-08-19 |
Gaucher Disease (1) · Hemophilia A (1)
Australia (1) · Brazil (1) · Canada (1) · France (1) · Germany (1) · Greece (1) · Italy (1) · Japan (1) · Saudi Arabia (1) · South Korea (1) · Spain (1) · Sweden (1) · Taiwan (1) · Turkey (Türkiye) (1) · United Kingdom (1)