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

ConditionsAdrenoleukodystrophy 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

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age1-22

SponsorJoanne Kurtzberg, MD

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 the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

Eligibility criteria

Qualifiers

Patients must be age ≥1 week to ≤21 years.

Abnormal EEG, Brainstem Auditory Evoked Response (BAER), and/or Visual Evoked Potentials (VEP).

Abnormal brain MRI, ie. increased Loes score (measure of white matter damage, demyelination, and brain atrophy) and/or abnormal corticospinal tracts as assessed by MRI with diffusion tensor imaging (DTI).

Three or more of the early clinical markers: problems sleeping, increased activity, behavior difficulties, seizure-like activity, chewing behavior, inappropriate bladder training, inappropriate bowel training.

Disqualifiers

Prior organ, tissue, or stem cell transplant within 3 years of study entry.

Prior participation in any gene or regenerative cell therapy study.

Inability to have an MRI scan or lumbar puncture.

Intractable seizures.

Trial design

Treatments tested in this trial

DUOC-01

Treatment groups

40 Participants

are divided into 1 treatment group

Sponsors and collaborators

Joanne Kurtzberg, MD

Lead sponsor

Duke University

Sponsor institution

Source ClinicalTrials.gov