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Treatment
Mozobil 20 mg/ml solution for injection clinical trials
3 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 - AuthorisedPhase I and Phase II (Integrated)- First administration to humans
A phase I/II open label study to assess safety, feasibility and efficacy of ex vivo expanded, autologous haematopoietic stem and progenitor cell populations that contain CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA in children with autosomal recessive osteopetrosis caused by mutations in the TCIRG1 gene.
This study is for children with a rare bone disease called osteopetrosis caused by a specific gene fault. It tests a new genetic cell therapy to see if it's safe, works, and improves their health by correcting the faulty gene in their own cells.
For: Autosomal recessive osteopetrosis caused by mutations in the TCIRG1 geneItaly