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Sponsored by: |
Children's Hospital Los Angeles |
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Information provided by: | Children's Hospital Los Angeles |
ClinicalTrials.gov Identifier: | NCT00730314 |
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cell include: Red blood cell defects e.g. hemoglobinopathies (sickle cell disease and thalassemia), Blackfan-Diamond anemia and congenital or chronic hemolytic anemias; White blood cells defects/immune deficiencies e.g. chronic granulomatous disease, Wiskott-Aldrich syndrome,Osteopetrosis, Kostmann's syndrome (congenital neutropenia), Hereditary Lymphohistiocytosis (HLH); Platelets defects e.g.Congenital amegakaryocytic thrombocytopenia; Metabolic/storage disorders e.g. leukodystrophies,mucopolysaccharidoses as Hurler disease;Stem cell defects e.g.reticular agenesis, among many other rare similar conditions.
The study treatment plan uses a new transplant treatment regimen that aims to try to decrease the acute toxicities and complications associated with the standard treatment plans and to improve outcome
The blood stem cells will be derived from either unrelated donor or unrelated umbilical cord blood.
Condition | Intervention | Phase |
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Red Blood Cell Defects Sickle Cell Disease Thalassemia Blackfan-Diamond Anemia Leukocyte Defects and Immune Deficiencies Hereditary Lymphohistiocytosis Chronic Granulomatous Disease Wiskott-Aldrich Syndrome Chediak Higashi Syndrome CD40 Ligand Deficiency Hyper IgM Syndrome Leucocytes Adhesion Defect Type 1 Osteopetrosis Congenital Neutropenia X-Linked Lymphoproliferative Disease Platelets Defects Congenital Amegakaryocytic Thrombocytopenia Metabolic and Storage Disorders Hurler Disease Leukodystrophies Niemann-Pick Disease Fucosidosis Stem Cell Defects Reticular Agenesis |
Procedure: Hematopoietic stem cell transplantation |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Parallel Assignment |
Official Title: | Phase I/II Trial Of Hematopoietic Stem Cell Transplant (HSCT) For Children With A Genetic Disease Of Blood Cells Without An HLA-Matched Sibling Donor |
Estimated Enrollment: | 25 |
Study Start Date: | August 2008 |
Estimated Primary Completion Date: | August 2011 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
Unrelated donor
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Procedure: Hematopoietic stem cell transplantation
hematopoietic stem cell transplantation conditioning regimen depending on graft source
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2: Experimental
Cord Blood
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Procedure: Hematopoietic stem cell transplantation
hematopoietic stem cell transplantation conditioning regimen depending on graft source
|
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. The stem cells will be derived from a 1) matched unrelated donor (MUD) or 2) unrelated umbilical cord blood (UCB). Patients will receive a novel conditioning regimen with Busulfan, Cytoxan and Fludarabine (Bu/Cy/Flu) and either Alemtuzumab (Campath 1H) for recipients of a MUD or rabbit Antithymocyte Globulin (rATG) for recipients of unrelated UCB prior to hematopoietic stem cell transplant (HSCT).
We hypothesize that reduced dosages of Cytoxan will decrease the acute toxicities associated with the standard chemotherapies of Busulfan and Cytoxan (i.e. sinusoidal obstructive syndrome (SOS), hemorrhagic cystitis and mucositis). And the addition of fludarabine to a conditioning regimen with myeloablative doses of Busulfan and reduced dosages of Cytoxan prior to HSCT will overcome the engraftment barrier posed by an intact immune system, which is seen in patients with a genetic disease.
Ages Eligible for Study: | up to 21 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Hisham Abdel-Azim, MD | 323-361-8556 | habdelazim@chla.usc.edu |
United States, California | |
Children Hospital Los Angeles | Recruiting |
Los Angeles, California, United States, 90027 |
Principal Investigator: | Hisham Abdel-Azim, MD | Childrens Hospital Los Angeles, University of Southern California |
Responsible Party: | Childrens Hospital Los Angeles, University of Southern California ( Hisham Abdel-Azim, MD ) |
Study ID Numbers: | CCI #07-00119, CHLA-#07-00119 |
Study First Received: | August 6, 2008 |
Last Updated: | August 7, 2008 |
ClinicalTrials.gov Identifier: | NCT00730314 |
Health Authority: | United States: Institutional Review Board |
unrelated BMT HSCT bone marrow transplantation sickle cell disease thalassemia CGD |
HLH Blackfan-Diamond anemia Hurler leukodystrophy LAD I Genetic diseases |
Pick Disease of the Brain Sphingolipidoses Frontotemporal dementia Severe congenital neutropenia Leukocyte Disorders Brain Diseases Sickle cell anemia Alpha-L-iduronidase deficiency Osteopetrosis Metabolism, Inborn Errors Mucopolysaccharidoses Wiskott-Aldrich Syndrome Hemorrhagic Disorders Thrombocytopenia Leucocyte adhesion defect |
Hyper IgM syndrome Hyperkinesis Brain Diseases, Metabolic, Inborn Chediak-Higashi syndrome Niemann-Pick Disease Delirium Purpura Speech Disorders Metabolic Diseases Immunoproliferative Disorders Hematologic Diseases Blood Coagulation Disorders Blood Platelet Disorders Aphasia Lysosomal Storage Diseases |
Phagocyte Bactericidal Dysfunction Reticuloendotheliosis Disease Immune System Diseases Lysosomal Storage Diseases, Nervous System Nervous System Diseases Mucinoses |
Osteosclerosis Pathologic Processes Blood Coagulation Disorders, Inherited Syndrome Dysgammaglobulinemia Histiocytosis, Non-Langerhans-Cell Carbohydrate Metabolism, Inborn Errors |