Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
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|ClinicalTrials.gov Identifier: NCT00152100|
Recruitment Status : Completed
First Posted : September 9, 2005
Last Update Posted : May 20, 2009
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 or disease||Intervention/treatment||Phase|
|Severe Combined Immunodeficiency||Procedure: Stem cell transplant Drug: Filgrastim, Alemtuzumab Device: Miltenyi CliniMACS||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome|
|Study Start Date :||February 2004|
|Actual Primary Completion Date :||August 2007|
|Actual Study Completion Date :||August 2007|
- To investigate safety issues related to use of haploidentical highly purified CD133+ hematopoietic cells in patients with SCID
- To study the effects (good and bad) of this procedure
- To learn if this procedure will result in normal immune function in children with SCID
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00152100
|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|