Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

St. Jude Children's Research Hospital (Other)
Overall Status
Completed ID
Duration (Months)
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or DiseaseIntervention/TreatmentPhase
  • Procedure: Stem cell transplant
  • Drug: Filgrastim, Alemtuzumab
  • Device: Miltenyi CliniMACS
Phase 1

Study Design

Study Type:
Actual Enrollment :
4 participants
Intervention Model:
Single Group Assignment
None (Open Label)
Primary Purpose:
Official Title:
Transplantation of Highly Purified Haploidentical CD133 Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Study Start Date :
Feb 1, 2004
Actual Primary Completion Date :
Aug 1, 2007
Actual Study Completion Date :
Aug 1, 2007

Outcome Measures

Primary Outcome Measures

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

  2. To study the effects (good and bad) of this procedure []

  3. To learn if this procedure will result in normal immune function in children with SCID []

Eligibility Criteria


Ages Eligible for Study:
N/A to 2 Years
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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

Contacts and Locations


SiteCityStateCountryPostal Code
1St. Jude Children's Research HospitalMemphisTennesseeUnited States38105

Sponsors and Collaborators

  • St. Jude Children's Research Hospital


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

Study Documents (Full-Text)

None provided.

More Information

Additional Information:


None provided.
Responsible Party:
, , Identifier:
Other Study ID Numbers:
First Posted:
Sep 9, 2005
Last Update Posted:
May 20, 2009
Last Verified:
May 1, 2009

Study Results

No Results Posted as of May 20, 2009