Status: Recruiting
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.
Participants needed: 40
Trial details
Phase: Phase 1Age: 1-22Biological sex: AllType: InterventionalSponsor: Joanne Kurtzberg, MDUpdated: Sep 8, 2025Locations: 1
Eligibility criteria
Patients must be age ≥1 week to ≤21 years. [+11]
Prior organ, tissue, or stem cell transplant within 3 years of study entry. [+11]