Could a common arthritis drug tame rare immune disease Flare-Ups?
NCT ID NCT05104723
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested the drug tofacitinib (Xeljanz) in 10 adults with chronic granulomatous disease (CGD) who had ongoing inflammation in the gut, skin, or lungs. Participants took the pill daily for 3 months to see if it was safe and could help control symptoms. The goal was to find a better option for people who don't respond well to current treatments.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- tofacitinib (Xeljanz)
- What this could lead to
- If it works, this could point toward a new treatment option for people with chronic granulomatous disease who have ongoing inflammation.
- What could go wrong
- This is a very small, early-phase study with only 10 participants, so results may not apply to everyone. Tofacitinib can increase infection risk, which is especially concerning for people with CGD.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
10 people
The number who actually took part.
- Started
-
Aug 2022
- Finished
-
May 2026
- Lead sponsor
-
A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 99 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: In order to be eligible to participate in this study, an individual must meet all of the following criteria: 1. Aged \>=18 years. 2. Enrolled on NIH study 93-I-0119. 3. Has a documented diagnosis of one or more of the following and is not controlled under current therapy (per investigator assessment): 1. Endoscopically diagnosed mild-to-severe CGD-related IBD. 2. Radiographic or PFT changes (DLCO\<60%, FEV1\<70%) consistent with CGD-related inflammatory lung disease. 3. Any inflammatory skin disease related to CGD (eg, hidradenitis suppurativa or granulomatous skin disease). 4. Able to provide informed consent. 5. 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: An individual who meets any of the following criteria will be excluded from participation in this study: 1. Known allergy or hypersensitivity to any component of the tofacitinib formulation. 2. Known allergy or hypersensitivity to any component of the acyclovir or valacyclovir formulation. 3. Active or latent tuberculosis. 4. Infection with hepatitis B or C, or HIV. 5. Active EBV infection. 6. History of GI perforation. 7. History of malignancy (except for nonmelanoma skin cancer). 8. Concomitant use of acetylsalicylic acid and/or NSAIDs that cannot be safely discontinued. 9. History of connective tissue disease. 10. End-stage renal disease or chronic kidney disease, defined as estimated glomerular filtration rate (eGFR) \<15 mL/min/1.73 m\^2. 11. Evidence of other invasive or systemic fungal, bacterial, or viral infections requiring therapy. 12. Pregnant. 13. Breastfeeding. 14. Current use of inhaled tobacco products, vaping products, inhaled cannabis, or other illicit inhaled drugs. 15. Current use of strong CYP3A4 inducer and unable to discontinue at least 14 days before beginning of tofacitinib regimen. 16. Concomitant medical condition that could interfere with study drug evaluation or that is a contraindication to the proposed investigational treatment based upon known agent safety profile or toxicities. 17. Any of the following laboratory abnormalities: 1. Alkaline phosphatase and either ALT or AST \>2.5 times the upper limit of normal (ULN). 2. Serum creatinine level \>5 mg/dL. 3. Absolute neutrophil count (ANC) \<1000 cells/microL. 4. Lymphocyte count \<500 cells/microL. 18. History of unprovoked deep vein thrombosis, pulmonary embolism, or other thrombotic events. 19. History of heart failure. 20. Current immobilization, ie, bed-bound and unable to ambulate. 21. Exposure to any investigational agent within the last 4 weeks. 22. Any other finding that, in the judgment of the investigator, would interfere with, or serve as a contraindication to, protocol adherence, assessment of safety or reactogenicity, or a participant s ability to give informed consent, or increase the risk of having an adverse outcome from participating in the study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New hope for CGD: stem cell transplant trial aims for a cure