Status:
TERMINATED
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Lead Sponsor:
Aldagen
Conditions:
Inherited Metabolic Diseases
Lysosomal Storage Disorders
Eligibility:
All Genders
Up to 16 years
Phase:
PHASE3
Brief Summary
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease. A portion of cord blood cells (ALD-101) will be separate...
Detailed Description
Subjects will be hospitalized and undergo high doses of chemotherapy which will destroy the child's normal cells including their bone marrow (which forms blood cells) in order to prepare their body fo...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
March 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2011
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT00654433
Start Date
March 1 2008
End Date
November 1 2011
Last Update
July 8 2014
Active Locations (3)
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1
Mattel Children's Hospital at UCLA
Los Angeles, California, United States, 90095
2
Mt. Sinai Medical Center
New York, New York, United States, 27705
3
Duke University
Durham, North Carolina, United States, 27705