Merilen
Sponsors
Rocket Pharmaceuticals Inc.
Conditions
Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cellsPyruvate Kinase DeficiencyPyruvate kinase deficiency
Phase 1
Long-Term Follow-Up (LTFU) for Gene Therapy of Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD
Active, not recruitingCTIS2022-501526-38-00
Start: 2023-06-06Target: 4Updated: 2025-04-22
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD
CompletedCTIS2024-511520-13-00
Start: 2019-11-04End: 2025-06-09Target: 3Updated: 2025-05-05