Neupogen 30 MU (0

DRUG2 trials

Sponsors

Rocket Pharmaceuticals Inc., Resolution Therapeutics Limited

Conditions

Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cellsLiver CirrhosisPyruvate Kinase Deficiency

Phase 1

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

Rocket Pharmaceuticals Inc.Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cells, Pyruvate Kinase Deficiency

Start: 2019-11-04End: 2025-06-09Target: 3Updated: 2025-05-05

An Open-label Phase 1/2 Multicentre Study to Evaluate the Safety, Tolerability and Efficacy of RTX001 Autologous Macrophages in Participants with Liver Cirrhosis who have Hepatic Decompensation (EMERALD)

RecruitingCTIS2024-516288-10-00

Resolution Therapeutics LimitedLiver Cirrhosis

Start: 2025-03-21Target: 8Updated: 2025-11-05