Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Severe Immunodeficiency Diseases Clinical Trial

— SIDS  

Official title:

Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00613561
• Other study ID #: SCT 0707
• Secondary ID: IRB # 2007-13271
• Status: Recruiting
• Phase: Phase 2
• First received: January 31, 2008
• Last updated: May 26, 2009
• Start date: December 2007
• Est. completion date: December 2017

Study information

• Verified date: May 2009
• Source: Ann & Robert H Lurie Children's Hospital of Chicago
• Contact: Morris Kletzel, MD
• Phone: 773-880-4562
• Email: mkletzel[@]childrensmemorial.org
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 25
• Est. completion date: December 2017
• Est. primary completion date: December 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 11 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Immunologic Deficiency Syndromes
• Severe Immunodeficiency Diseases

Intervention

Drug:
• Fludarabine, Busulfan, and Anti-Thymocyte Globulin
Patients on this study will receive 5 days of Fludarabine, 1 day of Busulfan Test Dose, 2 Days of Busulfan Regimen Dose, and 4 days of Anti-Thymocyte Globulin

Locations

Country	Name	City	State
United States	Children's Memorial Hospital	Chicago	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes
Secondary	To prospectively follow the natural course of severe immunodeficiency diseases after transplantation		5 years	Yes
Secondary	To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.		5 years	Yes