Phase 2 Study of Single IV Dose of Autologous CD34 Cells Enriched with Placenta-Derived Mitochondria for Treating Pearson Syndrome in Children and Adolescents

What this Trial Is About

Primary Mitochondrial diseases are a group of disorders caused by mutations in genes from nuclear DNA or mitochondrial DNA, affecting one or multiple organs. Pearson Syndrome is one such mitochondrial disease that can present with various symptoms and progresses over time. This Phase II open-label study evaluates the safety and therapeutic effects of MNV-201, a cell therapy developed by Minovia Therapeutics Ltd., designed to treat patients diagnosed with Pearson Syndrome. MNV-201 is made by enriching a participants own CD34 hematopoietic stem and progenitor cells with mitochondria derived from donated placental tissue. The study involves a single intravenous infusion of these enriched cells. Participants undergo mobilization and leukapheresis to collect their cells, which are then processed and infused back. The trial monitors treatment effects over months and includes follow-ups up to 24 months after treatment. Participants will be involved in regular assessments including monitoring treatment-related adverse events for 12 months and measuring height standard deviation scores SDS at 12 and 24 months, plus kidney function through calculated glomerular filtration rate slope over 24 months. The study includes evaluations of growth and safety, requiring participants to have prior growth and kidney function data for at least 12 months before treatment. Parent or guardian consent and ongoing cooperation with study visits and assessments are essential during the trial period.

Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)