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ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00654433
Recruitment Status : Terminated (Terminated - Sponsor Decision)
First Posted : April 8, 2008
Last Update Posted : July 8, 2014
Information provided by (Responsible Party):

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 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.

Condition or disease Intervention/treatment Phase
Inherited Metabolic Diseases Lysosomal Storage Disorders Peroxisomal Storage Diseases Inborn Errors of Metabolism Mucopolysaccharidosis Biological: ALD-101 Phase 3

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 for the umbilical cord blood transplant. The cord blood transplant is intended to rescue your child's bone marrow from the bad effects of the procedure. The child will receive 80% of a standard cord blood transplant followed by 20% supplemental stem cell called ALD-101.

The study will evaluate if these cells (ALD-101) will repopulate the bone marrow more rapidly after transplant. This would shorten the period of time that the transplanted subject would be at risk for infection and bleeding and would also decrease the number of red blood cell and platelet transfusions needed.

ALD-101 has been used as a supplement to cord blood transplant in twenty-eight children with genetic diseases and malignancy in one previous clinical study that is on-going.

The main purpose of this research study is to test whether a portion of cord blood cells that have been separated from a cord blood unit (ALD-101) will increase the speed at which normal levels of circulating blood cells are re-established after transplant. This is the experimental part of this study. ALD-101 is also being tested to see if it is safe.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism
Study Start Date : March 2008
Actual Primary Completion Date : July 2011
Actual Study Completion Date : November 2011

Arm Intervention/treatment
Experimental: I Biological: ALD-101
A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).

Primary Outcome Measures :
  1. To assess the efficacy of adjuvant therapy of ALD-101 in accelerating platelet engraftment in patients also receiving a standard unrelated UCBT for treatment of inherited metabolic diseases [ Time Frame: 180 Days ]

Secondary Outcome Measures :
  1. To assess the efficacy of ALD-101 in accelerating neutrophil engraftment [ Time Frame: 180 Days ]
  2. To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure. [ Time Frame: 180 Days ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00654433

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United States, California
Mattel Children's Hospital at UCLA
Los Angeles, California, United States, 90095
United States, New York
Mt. Sinai Medical Center
New York, New York, United States, 27705
United States, North Carolina
Duke University
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
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Study Director: James Hinson, MD Aldagen
Principal Investigator: Joanne Kurtzberg, MD Duke University
Additional Information:
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Responsible Party: Aldagen Identifier: NCT00654433    
Other Study ID Numbers: UCBT-002
First Posted: April 8, 2008    Key Record Dates
Last Update Posted: July 8, 2014
Last Verified: July 2014
Keywords provided by Aldagen:
Metachromatic Leukodystrophy
Hurler Syndrome
Hurler-Scheie Syndrome
Hunter Syndrome
Sanfilippo Syndrome
Maroteaux-Lamy Syndrome
Krabbe Disease
Globoid Leukodystrophy
Sandhoff Disease
Tay Sachs Disease
Pelizaeus Merzbacher
Niemann-Pick Disease A
Niemann-Pick Disease B
Inherited metabolic diseases
Inborn errors of metabolism
Lysosomal storage diseases
Peroxisomal storage diseases
Hematopoietic stem cell transplantation
Umbilical cord blood transplantation
Additional relevant MeSH terms:
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Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Carbohydrate Metabolism, Inborn Errors
Genetic Diseases, Inborn
Connective Tissue Diseases