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Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00152100
Recruitment Status : Completed
First Posted : September 9, 2005
Last Update Posted : May 20, 2009
Information provided by:
St. Jude Children's Research Hospital

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 disease Intervention/treatment Phase
Severe Combined Immunodeficiency Procedure: Stem cell transplant Drug: Filgrastim, Alemtuzumab Device: Miltenyi CliniMACS Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

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

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 2 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

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 identifier (NCT number): NCT00152100

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United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
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Principal Investigator: Kimberly Kasow, DO St. Jude Children's Research Hospital
Additional Information:
Layout table for additonal information Identifier: NCT00152100    
Other Study ID Numbers: ALSCID
First Posted: September 9, 2005    Key Record Dates
Last Update Posted: May 20, 2009
Last Verified: May 2009
Keywords provided by St. Jude Children's Research Hospital:
Severe combined immunodeficiency
Additional relevant MeSH terms:
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Severe Combined Immunodeficiency
Immunologic Deficiency Syndromes
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Antineoplastic Agents, Immunological
Antineoplastic Agents