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

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ClinicalTrials.gov Identifier: NCT02116764
Recruitment Status : Enrolling by invitation
First Posted : April 17, 2014
Last Update Posted : March 24, 2021
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) )

Study Description
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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.
Study Design
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Study Type : Observational
Actual Enrollment : 137 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, 2022
Estimated Study Completion Date : December 1, 2022

Resource links provided by the National Library of Medicine


Groups and Cohorts
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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


Outcome Measures
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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)


Eligibility Criteria
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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.
Contacts and Locations
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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


Locations
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United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
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)
More Information
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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: March 24, 2021
Last Verified: November 10, 2020
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) ):
Rare Diseases
Primary Immune Deficiency Treatment 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