Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

This study has been completed.
Sponsor:
Information provided by:
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00152100
First received: September 7, 2005
Last updated: May 19, 2009
Last verified: May 2009

September 7, 2005
May 19, 2009
February 2004
August 2007   (final data collection date for primary outcome measure)
  • 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
Same as current
Complete list of historical versions of study NCT00152100 on ClinicalTrials.gov Archive Site
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Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

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.

Not Provided
Interventional
Phase 1
Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Severe Combined Immunodeficiency
  • Procedure: Stem cell transplant
  • Drug: Filgrastim, Alemtuzumab
  • Device: Miltenyi CliniMACS
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
4
August 2007
August 2007   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patient with confirmed severe combined immunodeficiency
  • Two years of age or younger
  • A suitable matched sibling donor is not available

Exclusion Criteria:

  • An available matched sibling donor or a confirmed matched unrelated donor
  • Patients with DiGeorge syndrome, Zap70, MHC Class II deficiency, or cartilage-hair hypoplasia
  • Patients with a Lansky performance score of less than 10, evidence of HIV or a congenital rubella infection or a documented neoplasm
  • Patients in whom it is not possible to perform a peripheral blood cell harvest on a haploidentical family member
Both
up to 2 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00152100
ALSCID
No
Not Provided
St. Jude Children's Research Hospital
Not Provided
Principal Investigator: Kimberly Kasow, DO St. Jude Children's Research Hospital
St. Jude Children's Research Hospital
May 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP