Analysis of Patients Treated for Chroni… | Clinical Trial

Key information

Conditions

Chronic Granulomatous Disease Chronic Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Genetic Diseases, X-Linked Granulomatous Disease, Chronic Hematologic Diseases Hemic and Lymphatic Diseases Immune System Diseases Immunologic Deficiency Syndromes Leukocyte Disorders Pathologic Processes Pathological Conditions, Signs and Symptoms Phagocyte Bactericidal Dysfunction Rare Diseases

Age range

3 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

The primary objective of this protocol is to estimate the 1- year, 2-year and 3-year (and longer if possible) overall survival probabilities post-HCT of CGD subjects born on or after 1988 who receive HCTon or after 1995.

Time frame: 1, 2 or 3 year

Clinical outcomes (survival, infection, autoimmune disease, chimerism, GVHD)

Secondary outcomes

To compare overall survival from birth between patients born on or after 1988 who receive HCT on or after 1995 vs. those born on or after 1988 who receive conventional therapy, after adjusting for differences in year of birth and oxidase acti...

Time frame: 3 years post Transplant or Non Transplant

Clinical outcomes (survival, infection, autoimmune disease, chimerism, GVHD)

Sponsors and collaborators

Lead sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Nih

Registry information

Important dates

Study start: 2014
Primary completion: 2022
Study completion: 2022
First posted: Apr 17, 2014
Registry last updated: Jul 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed