NCT02254863 UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
| NCT ID | NCT02254863 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Joanne Kurtzberg, MD |
| Condition | Adrenoleukodystrophy |
| Study Type | INTERVENTIONAL |
| Enrollment | 40 participants |
| Start Date | 2014-09 |
| Primary Completion | 2026-10 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 40 participants in total. It began in 2014-09 with a primary completion date of 2026-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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
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
Contact & Investigator
Sydney Crane, RN
✉ cordbloodtherapyinfo@dm.duke.eduJoanne Kurtzberg, MD
PRINCIPAL INVESTIGATOR
Duke University
Frequently Asked Questions
Who can join the NCT02254863 clinical trial?
This trial is open to participants of all sexes, aged 1 Week or older, up to 22 Years, studying Adrenoleukodystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT02254863 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT02254863 currently recruiting?
Yes, NCT02254863 is actively recruiting participants. Contact the research team at cordbloodtherapyinfo@dm.duke.edu for enrollment information.
Where is the NCT02254863 trial being conducted?
This trial is being conducted at Durham, United States.
Who is sponsoring the NCT02254863 clinical trial?
NCT02254863 is sponsored by Joanne Kurtzberg, MD. The principal investigator is Joanne Kurtzberg, MD at Duke University. The trial plans to enroll 40 participants.