Clinical Trial: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

Brief Summary: This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of protocols used by participating institutions compared to a control non-HCT group receiving standard care. Investigators at multiple centers caring for patients with CGD in North America and 3 centers in Europe will participate. Patients with CGD will have been treated according to institutional practice and protocols. Investigators will enroll these patients as subjects in this protocol. This study will investigate which patients benefit most from HCT, and what types of transplants are optimal for patients with CGD, in the context of overall outcomes in CGD patients with and without transplant.

Detailed Summary: This study is a longitudinal and cross-sectional evaluation of patients with Chronic Granulomatous Disease (CGD) who received or are receiving hematopoietic cell transplantation (HCT) for their disease under a variety of protocols used by participating institutions compared to a control non-HCT group receiving standard care. Investigators at multiple centers caring for patients with CGD in North America and 3 centers in Europe will participate. Patients with CGD will have been treated according to institutional practice and protocols. Investigators will enroll these patients as subjects in this protocol. This study will investigate which patients benefit most from HCT, and what types of transplants are optimal for patients with CGD, in the context of overall outcomes in CGD patients with and without transplant.
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Current Primary Outcome: The primary endpoint in this study is overall survival [ Time Frame: Overall survival will be estimated at 1, 2 and 3 years (and longer, numbers permitting) after transplant ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Survival of HCT subjects vs. conventional therapy subjects, immune reconstitution, engraftment, andclinical outcomes such as infection, autoimmune disease and inflammatory complications, GvHD, growth, and quality of life. [ Time Frame: from birth, where all subjects transplanted and nontransplanted will have been born in or after 1988, to death or last contact. ]

Original Secondary Outcome: Survival of HCT subjects vs. conventional therapy subjects, immune reconstitution, engraftment, andclinical outcomes such as infection, autoimmune disease and inflammatory complications, GvHD, growth, and quality of life. [ Time Frame: For the comparison of HCT subjects with conventional therapy subjects, the time to this event is time from birth, where all subjects transplanted and nontransplanted will have been born in or after 1988, to death or last contact.subjects transplanted and ]

Information By: National Institutes of Health Clinical Center (CC)

Dates:
Date Received: April 15, 2014
Date Started: March 26, 2014
Date Completion: July 1, 2019
Last Updated: April 21, 2017
Last Verified: January 30, 2017