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

NCT02254863 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2025-09-08

No results posted yet for this study

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.

Conditions

  • Adrenoleukodystrophy
  • Batten Disease
  • Mucopolysaccharidosis II
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Neimann Pick Disease
  • Pelizaeus-Merzbacher Disease
  • Sandhoff Disease
  • Tay-Sachs Disease
  • Brain Diseases, Metabolic, Inborn
  • Alpha-Mannosidosis
  • Sanfilippo Mucopolysaccharidoses

Interventions

BIOLOGICAL

DUOC-01

Intrathecal administration of DUOC-01

Sponsors & Collaborators

  • Joanne Kurtzberg, MD

    lead OTHER

Principal Investigators

  • Joanne Kurtzberg, MD · Duke University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Week
Max Age
22 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-09-30
Primary Completion
2026-10-31
Completion
2026-10-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02254863 on ClinicalTrials.gov