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

Part of paid clinical trials in Durham, North Carolina.

Sponsor
Joanne Kurtzberg, MD
Study ID
NCT02254863
Phase
PHASE1
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
1 Week - 22 Years
Healthy Volunteers
Not accepted

Interventions

  • DUOC-01 — BIOLOGICAL
    Intrathecal administration of DUOC-01

Study Details

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.

Key Dates

Start date
Sep 30, 2014
Status verified
Sep 2025
Primary completion
Oct 31, 2026
Completion
Oct 31, 2026

Study Design

Enrollment
40 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Intrathecal administration of DUOC-01
    Administration of DUOC-01, given intrathecally, between day 26 and 28 post unrelated cord blood transplant

Primary Outcome Measure

Evaluate for Infusional Toxicity [ Time Frame: 24 hours after infusion ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Duke University Medical CenterDurhamNorth Carolina27705
Sydney Crane, RN
[email protected]
Joanne Kurtzberg, MD (PRINCIPAL_INVESTIGATOR)
Jessica Sun, MD (SUB_INVESTIGATOR)

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By research site
Duke University Medical Center· Durham, NC

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