Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Phase 2

• Conditions: Severe Immunodeficiency Diseases

• Registration Number: NCT00613561
• Lead Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-12
• Study First Submitted: 2008-01-31
• Study First Submitted QC: 2008-02-12
• Study First Posted: 2008-02-13
• Status Verified: 2009-05
• Last Update Submitted: 2009-05-26
• Last Update Posted: 2009-05-27
• Primary Completion: 2012-12
• Study Completion: 2017-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

• Must show one of the following diseases:

  • SCIDS
  • 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

• Adequate Liver Function

• Adequate Cardiac Function

• Adequate Pulmonary Function

• Adequate Performance Statue

• Adequate Venous Access

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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	5 years

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Children's Memorial Hospital; 🇺🇸 Chicago, Illinois, United States