Inborn Errors of Metabolism Clinical Trial
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
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.
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.
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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