Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

NCT00152100 | Completed Phase 1

• 1 other identifier: ALSCID
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Severe Combined Immunodeficiency

Keywords

Severe combined immunodeficiency; Transplantation; Haploidentical

Outcome Measures

Primary Outcomes (3)

• 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

Interventions

Stem cell transplant PROCEDURE

Filgrastim, Alemtuzumab DRUG

Miltenyi CliniMACS DEVICE

Eligibility Criteria

• Age: Up to 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital

MeSH Terms

Conditions

Severe Combined Immunodeficiency

Interventions

Stem Cell Transplantation; Filgrastim; Alemtuzumab

Condition Hierarchy (Ancestors)

Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins

Study Officials

• Kimberly Kasow, DO

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: September 7, 2005
• First Posted: September 9, 2005
• Study Start: February 1, 2004
• Primary Completion: August 1, 2007
• Study Completion: August 1, 2007
• Last Updated: May 20, 2009

Record last verified: 2009-05

Locations

US (1)