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NCT02116764 Clinical Trial

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

Chronic Granulomatous Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT02116764
Other study ID # 140091
Secondary ID 14-I-0091
Status Completed
Phase
First received
Last updated
Start date June 11, 2014
Est. completion date June 2, 2022

Study information
Verified date March 26, 2024
Source National Institutes of Health Clinical Center (CC)
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Completed
Enrollment 137
Est. completion date June 2, 2022
Est. primary completion date June 2, 2022
Accepts healthy volunteers No
Gender All
Age group 3 Years and older
Eligibility - 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.

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
United States National Institutes of Health Clinical Center Bethesda Maryland

Sponsors (1)
Lead Sponsor Collaborator
National Institute of Allergy and Infectious Diseases (NIAID)

Country where clinical trial is conducted

United States, 

References & Publications (3)

Gallin JI, Alling DW, Malech HL, Wesley R, Koziol D, Marciano B, Eisenstein EM, Turner ML, DeCarlo ES, Starling JM, Holland SM. Itraconazole to prevent fungal infections in chronic granulomatous disease. N Engl J Med. 2003 Jun 12;348(24):2416-22. doi: 10.1056/NEJMoa021931. — View Citation

Kobayashi S, Murayama S, Takanashi S, Takahashi K, Miyatsuka S, Fujita T, Ichinohe S, Koike Y, Kohagizawa T, Mori H, Deguchi Y, Higuchi K, Wakasugi H, Sato T, Wada Y, Nagata M, Okabe N, Tatsuzawa O. Clinical features and prognoses of 23 patients with chronic granulomatous disease followed for 21 years by a single hospital in Japan. Eur J Pediatr. 2008 Dec;167(12):1389-94. doi: 10.1007/s00431-008-0680-7. Epub 2008 Mar 12. — View Citation

Kuhns DB, Alvord WG, Heller T, Feld JJ, Pike KM, Marciano BE, Uzel G, DeRavin SS, Priel DA, Soule BP, Zarember KA, Malech HL, Holland SM, Gallin JI. Residual NADPH oxidase and survival in chronic granulomatous disease. N Engl J Med. 2010 Dec 30;363(27):2600-10. doi: 10.1056/NEJMoa1007097. — View Citation

Outcome
Type Measure Description Time frame Safety issue
Primary 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. Clinical outcomes (survival, infection, autoimmune disease, chimerism, GVHD) 1, 2 or 3 year
Secondary 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... Clinical outcomes (survival, infection, autoimmune disease, chimerism, GVHD) 3 years post Transplant or Non Transplant
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