Using umbilical cord blood cells to treat inherited metabolic diseases

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial aims to evaluate the safety and feasibility of administering DUOC-01, a type of oligodendrocyte-like cell derived from umbilical cord blood, through the intrathecal route in patients with inherited metabolic diseases who are also receiving standard umbilical cord blood transplantation. The study focuses on patients with early signs of demyelinating disease in the central nervous system, assessing both the safety of this new treatment and its efficacy as an adjunctive therapy. By potentially enhancing the engraftment of donor cells in the CNS, this approach seeks to improve neurological outcomes for affected patients.

Why it matters

Who can join: the full rules

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 Syndrome (MPS II) Krabbe disease (Globoid Leukodystrophy) Metachromatic Leukodystrophy (MLD) Niemann Pick disease type A or B Pelizaeus-Merzbacher disease (PMD) Sandhoff disease Tay Sachs disease. Alpha Mannosidosis Sanfilippo (MPS III)
3. Patients must have neurologic evidence of their disease, either clinically or via neuroimaging or neurophysiological testing. Examples of evidence of neurologic involvement include, but are not limited to the following:

   * 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.

Exclusion Criteria:

1. Prior organ, tissue, or stem cell transplant within 3 years of study entry.
2. Prior participation in any gene or regenerative cell therapy study.
3. Inability to have an MRI scan or lumbar puncture.
4. Intractable seizures.
5. Chronic aspiration.
6. Bleeding disorder.
7. Evidence of HIV infection or HIV positive serology.
8. Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction.
9. Inability to obtain patient's, parent's or legal guardian's consent.
10. Requirement of ventilatory support.
11. Pregnant or breastfeeding.
12. Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy

Where it's running

• Duke University Medical Center
  Durham, North Carolina, United States Recruiting

Study contacts

• Principal investigator: Joanne Kurtzberg, MD — Duke University
• Study coordinator: Sydney Crane, RN
• Email: cordbloodtherapyinfo@dm.duke.edu

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.