Actively Recruiting
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Led by Joanne Kurtzberg, MD · Updated on 2025-09-08
40
Participants Needed
1
Research Sites
630 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be age 61 week to 61 21 years.
- Patients must have one of the following inherited metabolic diseases confirmed by enzyme or mutation analysis: Adrenoleukodystrophy, Batten Disease, Hunter Syndrome (MPS II), Krabbe disease, Metachromatic Leukodystrophy, Niemann Pick disease type A or B, Pelizaeus-Merzbacher disease, Sandhoff disease, Tay Sachs disease, Alpha Mannosidosis, Sanfilippo (MPS III).
- Patients must have neurological evidence of their disease through clinical signs or tests such as abnormal EEG, BAER, VEP, brain MRI with increased Loes score or abnormal corticospinal tracts, or at least three early clinical markers like sleep problems, increased activity, behavior difficulties, seizure-like activity, chewing behavior, or inappropriate bladder or bowel training.
- Patients must have adequate organ function: renal serum creatinine 61 2.0 mg/dl; hepatic ALT/AST 61 5 times normal, bilirubin 61 2.0 mg/dl (exceptions apply); normal cardiac function by echocardiogram or radionuclide scan (80% of normal for age); pulmonary function with FVC, FEV1, DLCO 61 60% predicted or O2 saturation 70% on room air if unable to perform tests.
- Patients must have an available, suitably matched, banked umbilical cord blood unit for transplant.
- Patients must have a performance status of Lansky 61 40% or Karnofsky 61 40%.
- Patients must have a life expectancy of at least 6 months.
You will not qualify if you...
- Prior organ, tissue, or stem cell transplant within 3 years before study entry.
- Prior participation in any gene or regenerative cell therapy study.
- Inability to have an MRI scan or lumbar puncture.
- Intractable seizures.
- Chronic aspiration.
- Bleeding disorder.
- Evidence of HIV infection or positive HIV test.
- Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT treatment.
- Inability to obtain consent from patient or legal guardian.
- Requirement of ventilatory support.
- Pregnant or breastfeeding.
- Active cancer or receiving concurrent radiotherapy, immunosuppressive drugs, or chemotherapy.
AI-Screening
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Trial Site Locations
Total: 1 location
1
Duke University Medical Center
Durham, North Carolina, United States, 27705
Actively Recruiting
Research Team
S
Sydney Crane, RN
CONTACT
E
Erin Arbuckle
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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