All treatments
Treatment
Busulfan Fresenius Kabi 6 mg/ml concentrate for solution for infusion clinical trials
2 indexed studies · 0 currently recruiting
- AuthorisedTherapeutic confirmatory (Phase III)
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
This study looks at a new gene therapy, exa-cel, for people with severe sickle cell disease. It aims to see if a single treatment can stop painful sickle cell crises for a year or more. Researchers will check if it's safe and how well it helps patients.
For: Sickle cell anaemiaItaly · France - Ongoing, recruitingHuman Pharmacology (Phase I)- Other
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
This study is testing a new gene therapy for adults with Pyruvate Kinase Deficiency (PKD). It aims to see if the treatment is safe by using the patient's own modified cells to improve red blood cell health.
For: Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cells · Pyruvate Kinase DeficiencySpain