Inborn Errors of Metabolism Clinical Trial
— UCBT-002Official title:
A Phase III Trial of ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transplantation (UCBT) in Patients With Inborn Errors of Metabolism
Verified date | July 2014 |
Source | Aldagen |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 40 |
Est. completion date | November 2011 |
Est. primary completion date | July 2011 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 16 Years |
Eligibility |
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 |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Duke University | Durham | North Carolina |
United States | Mattel Children's Hospital at UCLA | Los Angeles | California |
United States | Mt. Sinai Medical Center | New York | New York |
Lead Sponsor | Collaborator |
---|---|
Aldagen |
United States,
Escolar ML, Poe MD, Martin HR, Kurtzberg J. A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation. Pediatrics. 2006 Sep;118(3):e879-89. Epub 2006 Aug 21. — View Citation
Gentry T, Deibert E, Foster SJ, Haley R, Kurtzberg J, Balber AE. Isolation of early hematopoietic cells, including megakaryocyte progenitors, in the ALDH-bright cell population of cryopreserved, banked UC blood. Cytotherapy. 2007;9(6):569-76. — View Citation
Martin PL, Carter SL, Kernan NA, Sahdev I, Wall D, Pietryga D, Wagner JE, Kurtzberg J. Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant. 2006 Feb;12(2):184-94. — View Citation
Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99-108. doi: 10.1038/sj.bmt.1705970. Epub 2008 Jan 7. Review. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | 180 Days | No | |
Secondary | To assess the efficacy of ALD-101 in accelerating neutrophil engraftment | 180 Days | No | |
Secondary | To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure. | 180 Days | Yes |
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