Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

Sponsor

St. Jude Children's Research Hospital (Other)

Overall Status

Completed

CT.gov ID

NCT00152100

Collaborator

(none)

Enrollment

Location

Duration (Months)

0.1

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

Study Design

Study Type:

Interventional

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 :

Feb 1, 2004

Actual Primary Completion Date :

Aug 1, 2007

Actual Study Completion Date :

Aug 1, 2007

Outcome Measures

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 []

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 2 Years

Sexes Eligible for Study:

All

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