Intrathecal DUOC-01 Cell Therapy to Support Umbilical Cord Blood Transplant in Children and Young Adults with Inherited Metabolic Brain Diseases

What this Trial Is About

Researchers are evaluating the safety and feasibility of giving DUOC-01, a cell therapy derived from umbilical cord blood, as an additional treatment for patients with inherited metabolic diseases IMD who show early signs of nerve damage in the central nervous system. These patients are also receiving standard treatment with unrelated umbilical cord blood transplantation UCBT. The study also aims to describe how effective the combined UCBT and DUOC-01 treatment is in these patients. IMDs are genetic disorders that often cause progressive neurological problems and early death due to enzyme defects and toxic buildup. The study involves administering DUOC-01 intrathecally directly into the spinal fluid between 26 and 28 days after the unrelated cord blood transplant. The DUOC-01 cells come from umbilical cord blood and may be from the same donor as the transplant or a second donor. This treatment is given as an adjunct to standard UCBT to help speed up the arrival of donor cells to the central nervous system and prevent disease progression while the transplant takes effect. Participants will be monitored for safety, including checking for toxic reactions 24 hours after the DUOC-01 infusion and neurological effects one month later. Researchers will also assess the treatments efficacy over 1 to 5 years. Evaluations include neurological exams, brain imaging, and tests of organ function. The study involves patients from 1 week to 21 years old with specific inherited metabolic disorders who have neurologic evidence of disease and are eligible for UCBT. The total duration of participation varies depending on follow-up assessments.

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells