Study of FT024 Gene-Modified Stem Cell Therapy for Children Under 2 Years with Autosomal Recessive Osteopetrosis Caused by TCIRG1 Mutations

What this Trial Is About

Researchers are evaluating FT024 gene-modified stem cell therapy in children aged 28 days to 2 years who have autosomal recessive osteopetrosis caused by TCIRG1 gene mutations. This phase IIIa open label study aims to assess the safety, feasibility, and effectiveness of this treatment in up to 8 children. The trial is conducted at a specialized pediatric center in Italy and includes participants who meet specific genetic and clinical criteria for this rare bone disorder. Participants undergo harvesting of their own hematopoietic stem and progenitor cells HSPCs, which are modified with the FT024 lentiviral vector to correct the genetic defect. After manufacturing in a GMP facility, patients receive a reduced toxicity conditioning regimen using Treosulfan and Thiotepa, followed by intravenous infusion of FT024. If needed, additional FT024 boost doses may be given within 180 days without further conditioning. Regular follow-up continues for 2 years, with an option for extended long-term monitoring up to 15 years. During the study, participants will have about 10 visits including assessments, hospital stays, and possible remote visits depending on their needs. Researchers will monitor overall survival, long-term engraftment, hematologic recovery, adverse events, and quality of life measures. The study lasts around 30 months per patient and includes detailed evaluations of blood parameters, transfusion needs, and genetic markers to understand the treatments impact. Safety and efficacy data will guide future development of this gene therapy.

Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)