An observational study in Chronic Granulomatous Disease and Inflammatory Bowel Disease, sponsored by National Institute of Allergy and Infectious Diseases (NIAID). Withdrawn at 1 site in United States. Open to participants aged 3 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2023-09-13.
Sponsored by National Institute of Allergy and Infectious Diseases (NIAID) · Observational
Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →
Among 31 studies in Granulomatous Disease, Chronic by National Institute of Allergy and Infectious Diseases (NIAID) — 7 open to participants now, 10 under way, none of them phase 3. Granulomatous Disease, Chronic · National Institute of Allergy and Infectious Diseases (NIAID).
Among the 31 studies in Granulomatous Disease, Chronic by National Institute of Allergy and Infectious Diseases (NIAID), this one is one of 2 that started in 2023, is one of 7 observational ones, and was the 21st registered.
79 studies on the registry are indexed under Granulomatous Disease, Chronic; 17 are open to participants now.
Browse Granulomatous Disease, Chronic studies →7 of the 31 studies in Granulomatous Disease, Chronic by National Institute of Allergy and Infectious Diseases (NIAID) are open now, in 1 country. Recruiting first, the newest first:
National Institute of Allergy and Infectious Diseases (NIAID) · Sites in United States
Ages 18–75 · Men only
National Institute of Allergy and Infectious Diseases (NIAID) · Sites in United States
Ages 18–75 · Men only
National Institute of Allergy and Infectious Diseases (NIAID) · Sites in United States
Ages 4–65
National Institute of Allergy and Infectious Diseases (NIAID) · Sites in United States
Healthy volunteers
National Institute of Allergy and Infectious Diseases (NIAID) · Sites in United States
Ages 18–70 · Healthy volunteers
By country, each count listing its studies:
Of the 12 Granulomatous Disease, Chronic studies recruiting now, 3 (25%) set no upper age limit and 9 (75%) stop at 75 or younger; 8 take children under 18; 2 take healthy volunteers.
This study: 3 Years and older · all sexes · healthy volunteers too.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
31 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 17 · Phase 2 11 · Phase 3 2 · Phase 4 2
First in 1991; this study’s year is marked. 2 started in 2023.
By drug class, each counting the studies that test any of its medicines:
Median enrollment 10 across the 24 interventional studies, 156 across the 7 observational. Of the 17 that completed or were terminated, 3 (18%) have results posted.
This tab as a report to print or save as PDF → With an account.
National Institute of Allergy and Infectious Diseases (NIAID) is the lead sponsor of 2,446 studies on the registry; 179 are open to participants now.
Of its 405 completed or terminated interventional studies of FDA-regulated products, 302 (75%) have results posted.
2 of the studies in Granulomatous Disease, Chronic by National Institute of Allergy and Infectious Diseases (NIAID) that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06253507National Institute of Allergy and Infectious Diseases (NIAID)pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD) | 2027-03-31 |
| NCT05189925National Institute of Allergy and Infectious Diseases (NIAID)NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD) | 2027-07-01 |
Add all 2 to your calendar (.ics) → With an account.
Among the 31 studies in Granulomatous Disease, Chronic by National Institute of Allergy and Infectious Diseases (NIAID), from their registry updates:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
Background:
PIDD stands for primary immune dysregulation. It is a general term that includes many different inherited immune system disorders. The immune system is the part of the body that helps fight disease and infection. People with PIDDs can develop many kinds of health problems. One of these is inflammatory bowel disease (IBD), which causes diarrhea and cramping. Researchers want to learn more about these disorders to develop possible treatments.
Objective:
To learn more about when and why IBD may develop in some people with PIDDs.
Eligibility:
People ages 3 and older who have PIDD or IBD.
Healthy volunteers in this age group are also needed.
Design:
Visit 1: Participants will be screened with physical exam, medical history, and blood and urine tests.
Visit 2: Participants will:
Participants will have 1 follow-up visit per year. They will repeat visit 2 procedures.
Participants will be contacted by phone or email in between yearly visits. They will be asked about their health. They will complete a quality-of-life questionnaire and send a stool sample that is collected at home.
If participants experience a sudden change in symptoms or undergo a new treatment, they may be asked to complete visit 2 procedures.
If participants are not able to come to NIH, study data and samples can be collected without an in-person visit.
Participants will have a final study visit about 10 years after Visit 1. They will repeat visit 2 procedures.
Over 1 million people suffer from IBD in the United States. Although the exact pathogenesis is unclear, IBD results from an inappropriate inflammatory response to intestinal microbes which is influenced by the environment in a genetically susceptible host. IBDs can be classified as conventional (Crohn disease (CD) and ulcerative colitis (UC)) and unclassified (early onset, difficult to treat, associated with monogenic disorders and PIDDs). Among the 200 IBD susceptibility loci identified in genome-wide association studies (GWAS), overlap with aberrations identified in PIDDs has been observed, thereby supporting the study of PIDDs to better understand conventional IBD pathogenesis, while recognizing PIDD-associated IBDs as distinct disease entities requiring specialized management.
The prevalence of PIDDs worldwide is estimated at 1 in 2000 live births and encompasses a growing list of over 300 PIDDs. Despite the fact that GI disease is the second most common complication in patients with PIDDs (rates ranging from 5-50%), little is known about PIDDspecific
IBD pathogenesis and even less is understood about the role of the microbiota both as a consequence and modulator of immune response in these inherited disorders. Moreover, there may be a time-limited period ("immunological window of opportunity"), coinciding with the maturation of the host s microbiome, during which early immune education may have long-term effects on predisposition to aberrant immune responses and inflammatory dysregulation. The primary objective of this study is to determine if PIDDs result in intestinal dysbioses, which alter local and systemic immune responses. Our long-term goal is to comprehensively investigate the immunological window of opportunity as it relates to PIDD-associated IBDs to define time-sensitive immunoregulatory targets for therapeutic intervention. We will pursue this goal through a prospective, longitudinal study of pediatric and adult patients with PIDDs (with and without IBD) before and after treatment and/or diagnostic interventions, including but not limited to hematopoietic stem cell transplantation (HSCT). Findings from subjects with PIDDs will be compared to those from subjects with IBD as well as healthy volunteers. This multifaceted study will complement primary patient protocols while allowing for the direct interrogation of specific arms of innate and adaptive immunity in the context of host-microbiome interactions. Patients will be studied over time through the collection of clinical metadata, blood, stool, urine, saliva, skin swabs, and biopsies obtained from clinically-indicated endoscopies in age-appropriate patients. PIDDs of interest include but are not limited to: CGD, CTLA4 and LRBA protein deficiency, hypomorphic RAG deficiency, and IPEX syndrome.
The study population will include patients and healthy volunteers greater than or equal to 3 years of age; PIDDs of interest, IBD, and Non-PIDD/non-IBD (healthy volunteers)@@@@@@
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
General Inclusion Criteria - All individuals must meet the following criteria to be eligible for study participation:
Specific Inclusion Criteria for IBD Cohort:
Specific Inclusion Criteria for non-PIDD/non-IBD Cohort (Healthy Volunteers):
EXCLUSION CRITERIA:
General Exclusion Criteria - An individual who meets any of the following criteria will be excluded from study participation:
Specific Exclusion Criteria for non-PIDD/non-IBD Cohort (Healthy Volunteers):
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
-Patients with IBD without a diagnosed PIDD.-First- or second-degree relatives of patients with a PIDD of interest who do not have a PIDD themselves, but have diagnosed or suspected IBD.
healthy volunteers
CGD cohort; IPEX syndrome cohort; CTLA4 haploinsufficiency cohort; LRBA deficiency and hypomorphic RAG deficiency cohorts
Disease-specific intestinal microbiome signatures, and related localized and systemic immune responses.
1. Microbiome, metabolomics, and transcriptomic signatures will be examined between comparison groups.
Time frame: Baseline and Annual follow-up visits spanning 10 years
Changes in microbiome signatures. Changes in metabolomic and transcriptomic signatures.
1. Microbiome, metabolomics, and transcriptomic signatures will be examined between comparison groups.
Time frame: Baseline and Annual follow-up visits spanning 10 years
Changes in systemic and tissue-specific markers of innate and adaptive immunity.
2. For evaluation of markers of innate and adaptive immunity (local and systemic) most comparisons will be made using 2-tailed Student t tests with statistical significance set at a p less than or equal to 0.05.
Time frame: Baseline and Annual follow-up visits spanning 10 years
Disease-specific differences in relative quantity and function of peripheral blood and tissue immune cells (includes hematopoietic and stromal cells).
3. For evaluation of disease-specific differences in relative quantity and function of peripheral blood and tissue immune cells most comparisons will be made using 2-tailed Student t tests with statistical significance set at a p less than or equal to 0.05.
Time frame: Throughout length of study
Plan to share: Yes — .The following will be shared:@@@@@@ (Summation)De-identified data in an NIH-funded or approved public repository.@@@@@@ (Summation)Identified data in the Biomedical Translational Research Information System (BTRIS).@@@@@@.De-identified or identified data with approved NIH-based and/or outside collaborators under appropriate agreements.
Supporting information: Statistical analysis plan
Time frame: Data will be shared before publication, at publication or shortly after publication.
Access criteria: Data will be shared through:@@@@@@ (Summation)An NIH-funded or approved public repository: National Center for Biotechnology Information Sequence Read Archive (NCBI SRA) Database.@@@@@@ (Summation)BTRIS.@@@@@@ (Summation)Approved outside collaborators under appropriate individual agreements.@@@@@@ (Summation)Publication and/or public presentations.@@@@@@The PI will review and approve/disapprove requests for IDP.
Ran from Sep 2023 to primary completion in Sep 2023, 0 months.
How these figures are worked out, and which studies are left out of them.
This study is withdrawn, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Granulomatous Disease, Chronic →
National Institute of Allergy and Infectious Diseases (NIAID) →