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RECRUITING
NCT02254863
PHASE1

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

Sponsor: Joanne Kurtzberg, MD

View on ClinicalTrials.gov

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.

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

Key Details

Gender

All

Age Range

1 Week - 22 Years

Study Type

INTERVENTIONAL

Enrollment

40

Start Date

2014-09

Completion Date

2026-10

Last Updated

2025-09-08

Healthy Volunteers

No

Interventions

BIOLOGICAL

DUOC-01

Intrathecal administration of DUOC-01

Locations (1)

Duke University Medical Center

Durham, North Carolina, United States