Health TrialsbyHealthPanel

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

ConditionsAdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis IILeukodystrophy, Globoid CellLeukodystrophy, MetachromaticNeimann Pick DiseasePelizaeus-Merzbacher DiseaseSandhoff DiseaseTay-Sachs DiseaseBrain Diseases, Metabolic, InbornAlpha-MannosidosisSanfilippo 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 in patients who are undergoing standard treatment with umbilical cord blood transplant (UCBT) for inborn errors of metabolism and who have evidence of early demyelinating disease in the central nervous system (CNS). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

Eligibility criteria

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • DUOC-01

    Biological/Vaccine

    Intrathecal administration of DUOC-01

Treatment groups

40 Participants
are divided into 1 treatment group
Group A: Intrathecal administration of DUOC-01Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Evaluate for Infusional Toxicity

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

Time frame
24 hours after infusion
2

Evaluate for Neuro Toxicity

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

Time frame
1 month after infusion

Secondary outcomes

1

Efficacy determination

Perform standard of care follow-up evaluations to include brain magnetic resonance imaging (MRI) with diffuse tensor imaging (DTI), Electroencephalography (EEG), nerve conduction, brainstem auditory evoked response (BAER), visual evoked potential (VEP) and neurocognitive testing. Bench mark results against historical controls previously transplanted by our institution for the past 20 years.

Time frame
1-5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Joanne Kurtzberg, MD

Lead sponsor

Duke University

Sponsor institution

Source ClinicalTrials.gov
Apply now