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Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

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

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

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • 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

Enrollment: 4
Study Start Date: February 2004
Study Completion Date: August 2007
Primary Completion Date: August 2007 (Final data collection date for primary outcome measure)

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00152100

United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Principal Investigator: Kimberly Kasow, DO St. Jude Children's Research Hospital
  More Information

Additional Information: Identifier: NCT00152100     History of Changes
Other Study ID Numbers: ALSCID
Study First Received: September 7, 2005
Last Updated: May 19, 2009

Keywords provided by St. Jude Children's Research Hospital:
Severe combined immunodeficiency

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Severe Combined Immunodeficiency
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases
Antineoplastic Agents processed this record on September 19, 2017