Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-02-29

Study Completion Date

2007-08-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Severe Combined Immunodeficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Stem cell transplant

Intervention Type PROCEDURE

Filgrastim, Alemtuzumab

Intervention Type DRUG

Miltenyi CliniMACS

Intervention Type DEVICE

Eligibility Criteria

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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

Maximum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No