Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

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Brief Title

Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

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.

Study Phase

Phase 1

Study Type


Primary Outcome

To investigate safety issues related to use of haploidentical highly purified CD133+ hematopoietic cells in patients with SCID


Severe Combined Immunodeficiency


Stem cell transplant


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

February 2004

Completion Date

August 2007

Primary Completion Date

August 2007

Eligibility Criteria

        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

          -  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




N/A - 2 Years

Accepts Healthy Volunteers



Kimberly Kasow, DO, , 

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Study Sponsor

St. Jude Children's Research Hospital

Study Sponsor

Kimberly Kasow, DO, Principal Investigator, St. Jude Children's Research Hospital

Verification Date

May 2009