Status:
RECRUITING
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Lead Sponsor:
Joanne Kurtzberg, MD
Conditions:
Adrenoleukodystrophy
Batten Disease
Eligibility:
All Genders
1-22 years
Phase:
PHASE1
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 ev...
Detailed Description
The inherited metabolic disorders (IMD) are a heterogeneous group of genetic diseases, most of which involve a single gene mutation resulting in an enzyme defect. In the majority of cases, the enzyme ...
Eligibility Criteria
Inclusion
- Patients must be age ≥1 week to ≤21 years.
- 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)
- 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.
- 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.
- Patients must have an available, suitably matched, banked UCB unit for transplant.
- Patients must have a performance status as follows: Lansky ≥ 40%, or Karnofsky ≥ 40%
- Patients must have a life expectancy of ≥ 6 months.
Exclusion
- 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.
- Chronic aspiration.
- Bleeding disorder.
- Evidence of HIV infection or HIV positive serology.
- Uncontrolled bacterial, viral, or fungal infection at the time of pre-UCBT cytoreduction.
- Inability to obtain patient's, parent's or legal guardian's consent.
- Requirement of ventilatory support.
- Pregnant or breastfeeding.
- Active concurrent malignancy, or receiving concurrent radiotherapy, immunosuppressive medications, or cytotoxic chemotherapy
Key Trial Info
Start Date :
September 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2026
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT02254863
Start Date
September 1 2014
End Date
October 1 2026
Last Update
September 8 2025
Active Locations (1)
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1
Duke University Medical Center
Durham, North Carolina, United States, 27705