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.
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