ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Stopped early · Phase 3

Conditions studied: Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis

In brief

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 separated from the cord blood unit and given approximately 4 hours after the standard cord blood transfusion. The study will test if the supplemental cells will increase the speed at which normal levels of circulating blood cells are re-established after transplant.

Key facts

Study ID

NCT00654433

Run by

Aldagen

People needed

40

Starts

2008-03-01

Expected to finish

2011-11-01

Last updated by the study team

2014-07-08

Who can join

Age: any, up to 16. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Mattel Children's Hospital at UCLA — Los Angeles, California, United States
• Mt. Sinai Medical Center — New York, New York, United States
• Duke University — Durham, North Carolina, United States

Full record on ClinicalTrials.gov

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