Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications (NCT05104723) | Clinical Trial Compass
CompletedPhase 1/2
Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications
United States10 participantsStarted 2022-08-12
Plain-language summary
Background:
Chronic granulomatous disease (CGD) is a disease of the immune system, which is how the body fights germs. People with CGD get infections easily and have other health problems. Some medicines to treat CGD have a lot of side effects and do not always work. Researchers want to see if a new drug can help.
Objective:
To see if tofacitinib is safe to use for treating chronic CGD.
Eligibility:
Adults aged 18 and older with CGD who have not had success with other treatments and who are enrolled on NIH study # 93-I-0119.
Design:
Participants will be screened with the following:
Physical exam
Medical history
Blood, urine, and stool tests
Pregnancy test, if needed
An upper gastrointestinal endoscopy and/or colonoscopy, if needed for their symptoms. Tissue samples will be collected.
Skin assessment, if needed
Participants will repeat some screening tests at visits.
Participants will complete questionnaires about their general health and how CGD affects their daily life. Photographs will be taken of their skin, if needed. They will have lung function tests, if needed. They will have a computed tomography (CT) scan of the chest, abdomen, and pelvis, if needed. A CT scan uses X-rays to create pictures of the inside of the body.
Participants will gradually reduce the amount of some CGD medicines they take. Then they will take tofacitinib as a pill twice a day or once a day for 3 months. They will keep a drug diary. They will have monthly study visits. They will have a follow-up visit about 1 month after their last study drug visit.
Participation will last for about 6 months....
Who can participate
Age range
18 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Aged \>=18 years.
. Enrolled on NIH study 93-I-0119.
. Has a documented diagnosis of one or more of the following and is not controlled under current therapy (per investigator assessment):
. Radiographic or PFT changes (DLCO\<60%, FEV1\<70%) consistent with CGD-related inflammatory lung disease.
. Any inflammatory skin disease related to CGD (eg, hidradenitis suppurativa or granulomatous skin disease).
. Able to provide informed consent.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Rate of treatment-related toxicities.
Timeframe: Day 1 through Day 120
2
Rate of infection.
Timeframe: Day 1 through Day 120
3
Rate of AEs
Timeframe: Day 1 through Day 120
4
Incidence of serious bacterial, mycobacterial, fungal, or viral infections defined as infections that require medical assessment or hospitalization.
Timeframe: Day 1 through Day 120
Trial details
NCT IDNCT05104723
SponsorNational Institute of Allergy and Infectious Diseases (NIAID)
. Participants who can become pregnant or who can impregnate their partner must agree to use at least one highly effective method of contraception when engaging in sexual activities that can result in pregnancy, starting at the first dose of tofacitinib until 2 days after the last dose. Highly effective methods include a barrier (eg, condom, diaphragm, cervical cap), intrauterine device, or hormonal contraception.
Exclusion criteria
. Known allergy or hypersensitivity to any component of the tofacitinib formulation.
. Known allergy or hypersensitivity to any component of the acyclovir or valacyclovir formulation.
. Active or latent tuberculosis.
. Infection with hepatitis B or C, or HIV.
. Active EBV infection.
. History of GI perforation.
. History of malignancy (except for nonmelanoma skin cancer).
. Concomitant use of acetylsalicylic acid and/or NSAIDs that cannot be safely discontinued.