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Sponsored by: |
St. Jude Children's Research Hospital |
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Information provided by: | St. Jude Children's Research Hospital |
ClinicalTrials.gov Identifier: | NCT00152100 |
Treatment for severe combined immunodeficiency (SCID) is a medical emergency. A stem cell transplant (immature blood cells that can make other blood cells) from a (MSD) matched sibling donor (brother or sister who is a "match" for your child's immune (HLA) type), usually results in complete correction of immune function.
However, most patients lack a matched sibling donor, requiring the use of an alternate donor source.
Transplantation of cells from haploidentical family donors (typically parents) has resulted in immune system correction in the majority of SCID individuals. However, only 65-80% of patients survive greater than one year after this procedure. Failure results from life-threatening infections, graft versus host disease (GvHD) or post-transplant treatment-related effects. Also, for patients that survive beyond one year, B-cell (type of blood cell that fights infection) and natural killer cell function (cell that attacks infections and cancer cells) frequently fail to work, resulting in the need for long-term treatment with intravenous gamma-globulin (IVIg).
In this study, in an effort to restore the overall cell function in patients with SCID, researchers will use a highly purified CD133+ hematopoietic cell graft (stem cell transplant without many mature donor white cells, called T-cells) obtained via use of the Miltenyi CliniMACS device, a device not FDA approved.
Condition | Intervention | Phase |
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Severe Combined Immunodeficiency |
Procedure: Stem cell transplant Drug: Filgrastim, Alemtuzumab Device: Miltenyi CliniMACS |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study |
Official Title: | Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome |
Enrollment: | 4 |
Study Start Date: | February 2004 |
Study Completion Date: | August 2007 |
Primary Completion Date: | August 2007 (Final data collection date for primary outcome measure) |
Ages Eligible for Study: | up to 2 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Tennessee | |
St. Jude Children's Research Hospital | |
Memphis, Tennessee, United States, 38105 |
Principal Investigator: | Kimberly Kasow, DO | St. Jude Children's Research Hospital |
Study ID Numbers: | ALSCID |
Study First Received: | September 7, 2005 |
Last Updated: | May 19, 2009 |
ClinicalTrials.gov Identifier: | NCT00152100 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Severe combined immunodeficiency Transplantation Haploidentical |
Metabolic Diseases Severe Combined Immunodeficiency Alemtuzumab |
Infant, Newborn, Diseases Metabolic Disorder Immunologic Deficiency Syndromes |
Metabolic Diseases Immune System Diseases Antineoplastic Agents Severe Combined Immunodeficiency Therapeutic Uses |
Alemtuzumab DNA Repair-Deficiency Disorders Infant, Newborn, Diseases Pharmacologic Actions Immunologic Deficiency Syndromes |