- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02116764
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
March 27, 2024 updated by: National Institute of Allergy and Infectious Diseases (NIAID)
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 Overview
Status
Completed
Conditions
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
Enrollment (Actual)
137
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Maryland
-
Bethesda, Maryland, United States, 20892
- National Institutes of Health Clinical Center
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
3 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Sampling Method
Probability Sample
Study Population
Patients diagnosed with CGD that will be or already Transplanted@@@@@@
Description
- 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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
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
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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)
|
1, 2 or 3 year
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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)
|
3 years post Transplant or Non Transplant
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Elizabeth M Kang, M.D., National Institute of Allergy and Infectious Diseases (NIAID)
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- 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.
- 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.
- 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.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 11, 2014
Primary Completion (Actual)
June 2, 2022
Study Completion (Actual)
June 2, 2022
Study Registration Dates
First Submitted
April 15, 2014
First Submitted That Met QC Criteria
April 15, 2014
First Posted (Estimated)
April 17, 2014
Study Record Updates
Last Update Posted (Actual)
March 28, 2024
Last Update Submitted That Met QC Criteria
March 27, 2024
Last Verified
March 26, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immunologic Deficiency Syndromes
- Immune System Diseases
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Disease Attributes
- Hematologic Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Leukocyte Disorders
- Phagocyte Bactericidal Dysfunction
- Chronic Disease
- Granuloma
- Granulomatous Disease, Chronic
Other Study ID Numbers
- 140091
- 14-I-0091
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Chronic Granulomatous Disease
-
National Institute of Allergy and Infectious Diseases...Enrolling by invitationChronic Granulomatous Disease (CGD) | X-Linked Chronic Granulomatous DiseaseUnited States
-
University of California, Los AngelesNational Heart, Lung, and Blood Institute (NHLBI); Boston Children's Hospital; California Institute for Regenerative Medicine (CIRM) and other collaboratorsRecruitingGranulomatous Disease, Chronic, X-linkedUnited States
-
National Institute of Allergy and Infectious Diseases...Active, not recruitingChronic Granulomatous Disease TransplantUnited States
-
GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseFrance
-
Children's Hospital of Fudan UniversityRecruitingBCG | Vitamin D3 | Chronic-granulomatous DiseaseChina
-
Great Ormond Street Hospital for Children NHS Foundation...WithdrawnGranulomatous Disease, Chronic, X-linked, VariantUnited Kingdom
-
GenethonActive, not recruitingX-Linked Chronic Granulomatous DiseaseUnited Kingdom
-
National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease-associated ColitisUnited States
-
National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...Enrolling by invitationGranulomatous Disease, ChronicUnited States, Canada
-
Hubert Serve, Prof., MDUnknown