ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases
About this trial
This is an interventional treatment trial for Inherited Metabolic Diseases focused on measuring Hurler Syndrome, MPS I, Hurler-Scheie Syndrome, Hunter Syndrome, MPS II, Sanfilippo Syndrome, MPS III, Maroteaux-Lamy Syndrome, MPS VI, Krabbe Disease, Globoid Leukodystrophy, Metachromatic Leukodystrophy, MLD, Adrenoleukodystrophy, ALD, Adrenomyeloneuropathy, AMN, Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher, PMD, Niemann-Pick Disease A, Niemann-Pick Disease B, Alpha-mannosidosis, Inherited metabolic diseases, Inborn errors of metabolism, Lysosomal storage diseases, Peroxisomal storage diseases, Hematopoietic stem cell transplantation, Umbilical cord blood transplantation, Mucopolysaccharidosis, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mucopolysaccharidosis VI, I Cell disease, Fucosidosis, GM1 Gangliosidosis, Canavan Disease
Eligibility Criteria
Inclusion Criteria:
confirmed diagnosis of inherited metabolic diseases; including the following:
- Hurler Syndrome (MPS I)
- Hurler-Scheie Syndrome
- Hunter Syndrome (MPS II)
- Sanfilippo Syndrome A and B(MPS III)
- Maroteaux-Lamy Syndrome (MPS VI)
- Krabbe Disease (Globoid Leukodystrophy)
- Metachromatic Leukodystrophy (MLD)
- Adrenoleukodystrophy (ALD and AMN)
- Sandhoff Disease
- Tay Sachs Disease
- Pelizaeus Merzbacher (PMD)
- Niemann-Pick Disease
- Alpha-mannosidosis
- I-Cell Disease (ML II)
- Fucosidosis
- GM I Gangliosidosis
- Canavan Disease
- must be <16 years of age at the time of study enrollment
- must have a good performance status (Lansky ≥80%)
- must have adequate function of other organ systems including: kidney, liver, heart and lungs
- must have given valid written informed consent
- must have a minimum life expectancy of at least 6 months
- must be determined to be a good candidate for a standard umbilical cord blood transplant
- must have an IQ >70 or if too young for IQ testing the potential to reach this endpoint by age 5
Exclusion Criteria:
- HIV, Hepatitis B and/or Hepatitis C positive
- concurrently involved in any other clinical study that affects engraftment or immune reconstitution
- uncontrolled seizures, apnea, evidence of aspiration pneumonia, or evidence of brain stem involvement
- uncontrolled infections
- prior allogeneic stem cell transplant with cytoreduction preparative therapy within 12 months of enrollment
Sites / Locations
- Mattel Children's Hospital at UCLA
- Mt. Sinai Medical Center
- Duke University
Arms of the Study
Arm 1
Experimental
I