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

National Institute of Allergy and Infectious Diseases (NIAID)

Status: Completed

Conditions: Chronic Granulomatous Disease

Study type: Observational

Funder types: NIH

Identifiers

NCT02116764

140091

14-I-0091

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 137 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

• Both HCT and non-HCT subjects must be over the age of 2 and actively enrolled and receiving treatment under a CGD protocol at NIAID.

Trial design

137 participants in 4 patient groups

Cross Sectional

Description:

3 years after Transplant

No Transplant

Description:

These patients were diagnosed with CGD but have not been transplanted

Prospective

Description:

Prior to Transplant Conditioning

Retrospective

Description:

1 year after Transplant