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| ID | Type | Description | Link |
|---|---|---|---|
| BB-IND#13502 |
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Terminated - Sponsor Decision
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| I | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALD-101 | Biological | A subpopulation of cord blood cells composed of cells that express a high level of the intracellular enzyme aldehyde dehydrogenase (ALDH). |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| To assess the efficacy of ALD-101 in accelerating neutrophil engraftment | 180 Days | |
| To assess the safety of adjuvant therapy of ALD-101 in infusional toxicity, adverse events, and primary graft failure. | 180 Days |
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Inclusion Criteria:
confirmed diagnosis of inherited metabolic diseases; including the following:
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:
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| Name | Affiliation | Role |
|---|---|---|
| James Hinson, MD | Aldagen | Study Director |
| Joanne Kurtzberg, MD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mattel Children's Hospital at UCLA | Los Angeles | California | 90095 | United States | ||
| Mt. Sinai Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 18176609 | Background | 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. | |
| 16443516 | Background | 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. doi: 10.1016/j.bbmt.2005.09.016. |
| Label | URL |
|---|---|
| Sponsor's Website | View source |
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| New York |
| New York |
| 27705 |
| United States |
| Duke University | Durham | North Carolina | 27705 | United States |
| 16923928 | Background | 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. doi: 10.1542/peds.2006-0747. Epub 2006 Aug 21. |
| 17882722 | Background | 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. doi: 10.1080/14653240701466347. |
| ID | Term |
|---|---|
| D016464 | Lysosomal Storage Diseases |
| D008661 | Metabolism, Inborn Errors |
| D009083 | Mucopolysaccharidoses |
| D008059 | Mucopolysaccharidosis I |
| D016532 | Mucopolysaccharidosis II |
| D013398 | Sudden Infant Death |
| D009084 | Mucopolysaccharidosis III |
| D009087 | Mucopolysaccharidosis VI |
| D007965 | Leukodystrophy, Globoid Cell |
| D007966 | Leukodystrophy, Metachromatic |
| D000326 | Adrenoleukodystrophy |
| D012497 | Sandhoff Disease |
| D013661 | Tay-Sachs Disease |
| D008363 | alpha-Mannosidosis |
| D009081 | Mucolipidoses |
| D005645 | Fucosidosis |
| D016537 | Gangliosidosis, GM1 |
| D017825 | Canavan Disease |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D003645 | Death, Sudden |
| D003643 | Death |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D066088 | Infant Death |
| D020279 | Hereditary Central Nervous System Demyelinating Diseases |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D056784 | Leukoencephalopathies |
| D003711 | Demyelinating Diseases |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D052439 | Lipid Metabolism Disorders |
| D052516 | Sulfatidosis |
| D018901 | Peroxisomal Disorders |
| D000309 | Adrenal Insufficiency |
| D000307 | Adrenal Gland Diseases |
| D004700 | Endocrine System Diseases |
| D020143 | Gangliosidoses, GM2 |
| D005733 | Gangliosidoses |
| D044904 | Mannosidase Deficiency Diseases |
| D001851 | Bone Diseases, Metabolic |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D019636 | Neurodegenerative Diseases |
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