Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Ann & Robert H Lurie Children's Hospital of Chicago1 site in 1 country25 target enrollmentDecember 2007

ConditionsSevere Immunodeficiency Diseases

DrugsFludarabine, Busulfan, and Anti-Thymocyte Globulin

Overview

Phase: Phase 2
Intervention: Not specified
Conditions: Severe Immunodeficiency Diseases
Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Enrollment: 25
Locations: 1
Primary Endpoint: To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation
Last Updated: 16 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.

Registry

clinicaltrials.gov

Start Date

December 2007

End Date

December 2017

Last Updated

16 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Eligibility Criteria

Inclusion Criteria

•Must show one of the following diseases:
•Hyper-IgM
•Wiskott-Aldrich Syndrome
•Chediak-Higashi and Griscelli Syndromes
•X-Linked Lymphoproliferative Diseases
•IPEX Syndrome
•NEMO Syndrome
•other severe immunodeficiency diseases not stated above at the discretion of the Principal Investigator
•Informed Consent
•Adequate Renal Function

Exclusion Criteria

•Patient/Family has not signed informed consent
•Patient does not have a clear diagnosis of a severe immunodeficiency disease
•A suitable donor for the patient cannot be found
•Patient is HIV positive
•Patient has active Hepatitis B
•Patient is pregnant
•Patient is considered unsuitable for transplant at the discretion of the Principal Investigators or the medical director

Outcomes

Primary Outcomes

To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation

Time Frame: 5 years

Secondary Outcomes

To prospectively follow the natural course of severe immunodeficiency diseases after transplantation(5 years)
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.(5 years)