Busulfan Fresenius Kabi 6 mg/ml concentrate for solution for infusion

DRUG3 trials

Sponsors

Vertex Pharmaceuticals Inc., Vaestra Goetalandsregionen, Rocket Pharmaceuticals Inc.

Conditions

Acute Myeoloid LeukemiaInherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cellsPyruvate Kinase DeficiencySickle cell anaemia

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

Phase 3

A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype

Not yet recruitingCTIS2023-503247-34-00

Vertex Pharmaceuticals Inc.Sickle cell anaemia

Target: 5Updated: 2025-08-19

A Randomized, Multi-Center Phase III Trial comparing two conditioning regimens (CloFluBu and BuCyMel) in children with Acute Myeloid Leukemia undergoing allogeneic stem cell transplantation.

RecruitingCTIS2023-505512-37-00

Vaestra GoetalandsregionenAcute Myeoloid Leukemia

Start: 2022-06-07Target: 105Updated: 2025-11-06