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

Recruiting Phase 1 Interventional Study
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
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
1 Week – 22
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
40 (estimated)
Sponsor
Joanne Kurtzberg, MD · Other
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About This Trial
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 t…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. Patients must be age ≥1 week to ≤21 years. 2. Patients must have one of the following inherited metabolic diseases detected by enzyme or mutation analysis, and confirmed by repeat testing on a separately obtained sample: Adrenoleukodystrophy (ALD) Batten Disease Hunter Sy…
Contacts

Sydney Crane, RN

cordbloodtherapyinfo@dm.duke.edu

CONTACT

Erin Arbuckle

cordbloodtherapyinfo@dm.duke.edu

CONTACT