UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells — DUOC-01  

Mucopolysaccharidosis II Clinical Trial

Official title: Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

Status Recruiting

Clinical Trial Summary

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.

Clinical Trial Description

The inherited metabolic disorders (IMD) are a heterogeneous group of genetic diseases, most of which involve a single gene mutation resulting in an enzyme defect. In the majority of cases, the enzyme defect leads to the accumulation of substrates that are toxic and/or interfere with normal cellular function. Often times, patients may appear normal at birth but during infancy begin to exhibit disease manifestations, frequently including progressive neurological deterioration due to absent or abnormal brain myelination. The ultimate result is death in later infancy or childhood. Currently, the only effective therapy to halt the neurologic progression of disease is allogeneic hematopoietic stem cell transplantation (HSCT), which serves as a source of permanent cellular ERT.3 However, one barrier to the success of this therapy is delayed engraftment of donor cells in the CNS when administered through the intravenous route, which is associated with ongoing disease progression over 2-4 months before stabilization. The engraftment of donor cells in a patient with an IMD provides a constant source of enzyme replacement, thereby slowing or halting the progression of disease. This study will evaluate the safety of a potential new treatment for patients with certain IMDs known to benefit from HSCT using allogeneic UCB donor cells. The new intervention, intrathecal administration of UCB-derived oligodendrocyte-like cells (DUOC-01) will serve as an adjunctive therapy to a standard UCB transplant. The goal of this therapy is to accelerate delivery of donor cells to the CNS thereby bridging the gap between systemic transplant and engraftment of cells in the CNS and preventing disease progression. The DUOC-01 cells and cells used for HSCT may be derived from the same UCB donor unit, or a second UCB donor unit will be used to manufacture the DUOC-01 cells. ;

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Alpha-Mannosidosis
• Batten Disease
• Brain Diseases
• Brain Diseases, Metabolic, Inborn
• Leukodystrophy, Globoid Cell
• Leukodystrophy, Metachromatic
• Metabolic Diseases
• Mucopolysaccharidoses
• Mucopolysaccharidosis II
• Neimann Pick Disease
• Neuronal Ceroid-Lipofuscinoses
• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• Pelizaeus-Merzbacher Disease
• Pick Disease of the Brain
• Sandhoff Disease
• Sanfilippo Mucopolysaccharidoses
• Tay-Sachs Disease

• NCT number: NCT02254863
• Study type: Interventional
• Source: Duke University
• Contact: Sydney Crane, RN
• Email: cordbloodtherapyinfo@dm.duke.edu
• Status: Recruiting
• Phase: Phase 1
• Start date: September 2014
• Completion date: October 2024