Clinical Trial: Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

Brief Summary:

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.


Detailed Summary:
Sponsor: St. Jude Children's Research Hospital

Current Primary Outcome:

  • 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


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: St. Jude Children's Research Hospital

Dates:
Date Received: September 7, 2005
Date Started: February 2004
Date Completion:
Last Updated: May 19, 2009
Last Verified: May 2009