ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)
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.
Inherited Metabolic Diseases
Lysosomal Storage Disease
Peroxisomal Storage Diseases
Inborn Errors of Metabolism
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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|
- 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 ] [ Designated as safety issue: No ]
- To assess the efficacy of ALD-101 in accelerating neutrophil engraftment [ Time Frame: 180 Days ] [ Designated as safety issue: No ]
- To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure. [ Time Frame: 180 Days ] [ Designated as safety issue: Yes ]
|Study Start Date:||March 2008|
|Estimated Study Completion Date:||November 2011|
|Estimated Primary Completion Date:||July 2011 (Final data collection date for primary outcome measure)|
A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH).
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.
|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|
|Durham, North Carolina, United States, 27705|
|Study Director:||James Hinson, MD||Aldagen|
|Principal Investigator:||Joanne Kurtzberg, MD||Duke University|