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Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995

Disease Information

Descriptive Information
Brief Title † Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995
Official Title † Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
Brief Summary Chronic granulomatous disease (CGD) is an inherited immune system abnormality in which bone marrow transplantation has been shown to be curative. However the risks of transplantation are high and not all patients with CGD may need to undergo this high risk procedure. This study will determine the long term medical condition and daily functioning of patients with CGD after a transplant and if possible, compare these results to patients who do not undergo a transplant. This multi-center study combines a longitudinal and cross-sectional evaluation (both retro and prospectively) of subjects with confirmed CGD who have already received hematopoietic cell transplant (HCT) since 1995 or who will receive HCT during the study period. This study will investigate which subjects benefit most from HCT, and the overall outcomes in CGD patients with and without transplant. The study aims to identify variables contributing to best outcomes of HCT in subjects with CGD.
Detailed Description
Study Phase N/A
Study Type † Observational
Study Design † Observational Model: Case Control
Primary Outcome Measure † Death
Secondary Outcome Measure † Engraftment
Condition † Granulomatous Disease, Chronic
Intervention †
Study Arms / Comparison Groups Longitudinal analysis: Retrospective CGD Cohort Longitudinal analysis: Prospective CGD Cohort Cross-sectional Analysis: HCT CGD Cohort Longitudinal Analysis: Conventional Non-Transplant CGD Cohort
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status †
Estimated Enrollment † 1480
Start Date † June 2014
Completion Date August 2019
Primary Completion Date August 2019
Eligibility Criteria † Inclusion Criteria: - Participant Inclusion Criteria (Part 1 - Longitudinal Analysis) - CGD Patients Undergoing Transplant 1995 to Present with Birth Year In or After 1988 1. CGD Patients will be Defined by both Defective Neutrophil NADPH Oxidase Function and by Clinical History Consistent with CGD Patients must have BOTH of: A functional assay demonstrating abnormal NADPH oxidase function (see A below); AND Clinical history consistent with CGD (see B below). ************************************************************************* Patients must have BOTH "A" and "B": A. Function: Assays of NADPH Oxidase Function I. Dihydrorhodamine (DHR) Assay: - Blood sample was obtained at a time when patient was clinically stable and not critically ill, with control samples performed simultaneously indicating a qualified assay; and - Assay unequivocally demonstrates CGD with an SI 2.5 will be classified as oxidase positive CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR - Ferricytochrome C reduction assay of granulocytes with O2 3 years post-diagnosis of CGD. Provision of written informed consent will be required for inclusion in the Cross-Sectional Analysis. Exclusion Criteria: - Participant Exclusion Criteria (Longitudinal and Cross- Sectional Analyses) - Presence of other primary immunodeficiency syndromes that do not meet the clinical and laboratory criteria for CGD. - Rac2 Deficiency - MPO Deficiency - Glutathione deficiency - Leukocyte adhesion deficiency syndrome - Non-transplant subjects: - The above exclusions pertain. - In addition, non-transplant subjects will be excluded if the only assessment of oxidase function available is the nitroblue tetrazolium (NBT) test (a non-quantitative test).
Gender Both
Ages N/A - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT02082353
Organization ID DAIT RDCRN PIDTC-6903
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † National Institute of Allergy and Infectious Diseases (NIAID)
Collaborators †† Primary Immune Deficiency Treatment Consortium
Investigators † Principal Investigator: Elizabeth M. Kang, MD, National Institute of Allergy and Infectious Diseases (NIAID)
Information Provided By
Verification Date June 2015
First Received Date † March 6, 2014
Last Updated Date June 2, 2015
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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