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Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome

NCT00152100 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2009-05-20

No results posted yet for this study

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

Interventions

PROCEDURE

Stem cell transplant

DRUG

Filgrastim, Alemtuzumab

DEVICE

Miltenyi CliniMACS

Sponsors & Collaborators

Principal Investigators

  • Kimberly Kasow, DO · St. Jude Children's Research Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-02-29
Primary Completion
2007-08-31
Completion
2007-08-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00152100 on ClinicalTrials.gov