Clinical Trial Details
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NCT02254863 : UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
PhasePhase 1
AgesMin: N/A Max: 22 Years
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

- 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.

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 not have a suitable fully matched, non-carrier sibling or related bone
marrow donor.

6. Patients must have an available, suitably matched, banked UCB unit in a
two-compartment configuration (see graft selection criteria in section 5.2).

7. Patients must have a performance status as follows: Lansky ? 40%, or Karnofsky ? 40%.

8. 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

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
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