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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) )

Tracking Information
First Submitted Date April 15, 2014
First Posted Date April 17, 2014
Last Update Posted Date September 23, 2019
Actual Study Start Date June 11, 2014
Estimated Primary Completion Date December 1, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 20, 2019)
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)
Original Primary Outcome Measures
 (submitted: April 15, 2014)
The primary endpoint in this study is overall survival [ Time Frame: Overall survival will be estimated at 1, 2 and 3 years (and longer, numbers permitting) after transplant ]
Change History
Current Secondary Outcome Measures
 (submitted: September 20, 2019)
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)
Original Secondary Outcome Measures
 (submitted: April 15, 2014)
Survival of HCT subjects vs. conventional therapy subjects, immune reconstitution, engraftment, andclinical outcomes such as infection, autoimmune disease and inflammatory complications, GvHD, growth, and quality of life. [ Time Frame: For the comparison of HCT subjects with conventional therapy subjects, the time to this event is time from birth, where all subjects transplanted and nontransplanted will have been born in or after 1988, to death or last contact.subjects transplanted and ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
Official Title Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
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.
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 Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population Patients diagnosed with CGD that will be or already Transplanted@@@@@@
Condition Chronic Granulomatous Disease
Intervention Not Provided
Study Groups/Cohorts
  • 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
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 24, 2018)
140
Original Estimated Enrollment
 (submitted: April 15, 2014)
120
Estimated Study Completion Date December 1, 2021
Estimated Primary Completion Date December 1, 2021   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 3 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Pamela D Graham (301) 761-6732 edmondsp@mail.nih.gov
Contact: Elizabeth M Kang, M.D. (301) 402-7567 ekang@niaid.nih.gov
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02116764
Other Study ID Numbers 140091
14-I-0091
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Institutes of Health Clinical Center (CC) ( National Institute of Allergy and Infectious Diseases (NIAID) )
Study Sponsor National Institute of Allergy and Infectious Diseases (NIAID)
Collaborators Not Provided
Investigators
Principal Investigator: Elizabeth M Kang, M.D. National Institute of Allergy and Infectious Diseases (NIAID)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date September 18, 2019