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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02116764
Recruitment Status : Recruiting
First Posted : April 17, 2014
Last Update Posted : September 23, 2019
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) )

Brief 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.

Condition or disease
Chronic Granulomatous Disease

Detailed 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.

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Study Type : Observational
Estimated Enrollment : 140 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
Actual Study Start Date : June 11, 2014
Estimated Primary Completion Date : December 1, 2021
Estimated Study Completion Date : December 1, 2021


Group/Cohort
Cross Sectional
3 years after Transplant
No Transplant
These patients were diagnosed with CGD but have not been transplanted
Prospective
Prior to Transplant Conditioning
Retrospective
1 year after Transplant



Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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)



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients diagnosed with CGD that will be or already Transplanted@@@@@@
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02116764


Contacts
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Contact: Pamela D Graham (301) 761-6732 edmondsp@mail.nih.gov
Contact: Elizabeth M Kang, M.D. (301) 402-7567 ekang@niaid.nih.gov

Locations
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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)    800-411-1222 ext TTY8664111010    prpl@cc.nih.gov   
Sponsors and Collaborators
National Institute of Allergy and Infectious Diseases (NIAID)
Investigators
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Principal Investigator: Elizabeth M Kang, M.D. National Institute of Allergy and Infectious Diseases (NIAID)

Additional Information:
Publications:
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Responsible Party: National Institute of Allergy and Infectious Diseases (NIAID)
ClinicalTrials.gov Identifier: NCT02116764    
Other Study ID Numbers: 140091
14-I-0091
First Posted: April 17, 2014    Key Record Dates
Last Update Posted: September 23, 2019
Last Verified: September 18, 2019
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) ):
Rare Diseases
Primary Immune Deficiency Consortium
Retrospective
Additional relevant MeSH terms:
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Granuloma
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases