Fancalen
Sponsors
Rocket Pharmaceuticals Inc.
Conditions
Fanconi anemia (subtype A)Fanconi anemia subtype A (FA-A)
Phase 1
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
RecruitingCTIS2022-501082-52-00
Start: 2023-03-01Target: 3Updated: 2025-05-08
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) in patients with Fanconi Anaemia Subtype A: FANCOLEN-I
RecruitingCTIS2024-511523-33-00
Start: 2020-05-28Target: 9Updated: 2025-06-23