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UCB Transplant with DUOC-01 for Inherited Metabolic Diseases

This study is testing a potential new treatment called DUOC-01 for children and young adults with certain inherited metabolic diseases, including Adrenoleukodystrophy, Batten Disease, and others. These diseases can cause serious brain problems. DUOC-01 is made from umbilical cord blood and is given directly into the fluid around the brain and spinal cord (intrathecal administration). This treatment is given along with a standard umbilical cord blood transplant. The main goals are to see if DUOC-01 is safe and practical, by checking for any side effects from the infusion within 24 hours and any brain-related side effects after one month. The study also aims to see how well this combined treatment works. You may be eligible if you are between 1 week and 22 years old and have one of these specific inherited metabolic diseases confirmed by testing. The current status of this study is unclear.

Study design
This is an interventional study planning to enroll 40 participants. It is not specified if it is randomized or blinded.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Safety will be evaluated for infusional toxicity at 24 hours after infusion and for neurotoxicity at 1 month after infusion.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT02254863

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

Recruiting
PHASE1Ages 1–22InterventionalTreatment
Joanne Kurtzberg, MD
~40 participants
Updated 2025-09-08 on ClinicalTrials.gov
What's tested:DUOC-01

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Evaluate for Infusional Toxicity
Measured over 24 hours after infusion
+1 more outcome measured

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

Explore related trials

AdrenoleukodystrophyAlpha-MannosidosisCongenital Metabolic DisorderHunter SyndromeKrabbe DiseaseMetachromatic LeukodystrophyNeuronal Ceroid Lipofuscinosis Type 3Niemann-Pick DiseasePelizaeus-Merzbacher DiseaseSandhoff DiseaseSanfilippo SyndromeTay-Sachs Disease

Where it's being run

1 sites across 1 states
North Carolina1

Study leadership

  • Joanne Kurtzberg, MD · PRINCIPAL_INVESTIGATOR · Duke University

Who to contact

Sydney Crane, RN
[email protected]
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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. 4. Patients must have adequate organ function as measured by:
Renal: Serum creatinine ≤ 2.0 mg/dl
Hepatic: Hepatic transaminases (ALT/AST) ≤ 5 x normal, bilirubin ≤ 2.0 mg/dl (except in patients with Gilbert's disease or newborns with physiological or breast milk associated jaundice).
Cardiac: Normal cardiac function by echocardiogram or radionuclide scan (shortening fraction or ejection fraction
80% of normal value for age). Patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide.
Pulmonary: Pulmonary function tests demonstrating FVC, FEV1, and DLCO ≥ 60% of predicted in patients who can complete the testing. If patient cannot perform PFT's, an O2 sat must be \>90% on room air. 5. Patients must have an available, suitably matched, banked UCB unit for transplant. 6. Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40% 7. Patients must have a life expectancy of ≥ 6 months.

What this trial measures

  • Evaluate for Infusional Toxicity24 hours after infusion

    Will monitor for fever, vomiting, neck stiffness, seizures, changes in state of consciousness

  • Evaluate for Neuro Toxicity1 month after infusion

    Perform computerized tomography (CT) scan to evaluate for bleeding, tumor formation, central nervous system generalized infiltration