Analysis of Autoinflammation in Chronic Granulomatous Disease Patients Undergoing Hematopoietic Cell Transplantation or Gene Therapy
A Study of CGD Autoinflammation PIDTC 6908
What is the goal of the study?
In this prospective longitudinal study, the aim is to determine if treatment with HCT /GT results in improvement or full resolution of CGD inflammatory disease, characterize the impact of inflammatory disease on HCT / GT outcomes, and identify optimal HCT / GT strategies for CGD patients both with and without inflammatory disease. The secondary aim is to identify in vitro biomarkers and microbiome signatures that correlate with the presence, severity, and control of inflammatory disease, HCT / GT outcomes and overall survival, and may be used as predictive indices to guide clinical management.For this prospective observational study, the treatment of inflammatory disease and other manifestations of CGD, HCT / GT strategy, and the management of post-HCT / GT complications will be determined by each treatment center. It is anticipated that differing HCT / GT approaches will be used at individual institutions. HCT / GT procedures and clinical assessments will be performed according to the prospective treatment protocols and practices of the individual participating sites. For this PIDTC natural history protocol, a standard dataset consisting of selected key information about the individual will be contributed by the participating site to the PIDTC Protocol 6908 dataset. Specific in vitro and microbiome studies will also be performed for the express purpose of this study.
Who can participate in the study?
Please contact the study team listed below to learn more.