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Janus Kinase (JAK) Inhibitor

Tofacitinib for Chronic Granulomatous Disease

Phase 1 & 2
Waitlist Available
Led By Christa S Zerbe, M.D.
Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Aged >=18 years
Has a documented diagnosis of endoscopically diagnosed mild-to-severe CGD-related IBD, radiographic or PFT changes consistent with CGD-related inflammatory lung disease, or any inflammatory skin disease related to CGD
Screening 3 weeks
Treatment Varies
Follow Up day 1 through day 120
Awards & highlights

Study Summary

This trial is testing a new drug to see if it's safe to use for treating chronic CGD, a disease of the immune system.

Who is the study for?
Adults over 18 with Chronic Granulomatous Disease (CGD) who've had little success with other treatments can join this trial. They must be part of NIH study #93-I-0119, not have severe kidney disease or infections like hepatitis B/C, HIV, or active EBV. Pregnant individuals and those using certain drugs are excluded.Check my eligibility
What is being tested?
The trial is testing Tofacitinib's safety and effectiveness for treating CGD complications. Participants will reduce current CGD meds and take Tofacitinib pills twice daily for three months while undergoing health assessments, including blood tests and CT scans.See study design
What are the potential side effects?
Tofacitinib may cause side effects such as increased risk of infections due to immune system suppression, potential liver issues indicated by blood test changes, possible kidney problems, and a higher chance of developing certain skin conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
I am 18 years old or older.
I have IBD, lung, or skin disease related to CGD diagnosed through endoscopy, imaging, or PFTs.


Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 through day 120
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 through day 120 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of serious bacterial, mycobacterial, fungal, or viral infections defined as infections that require medical assessment or hospitalization.
Rate of AEs
Rate of infection.
+1 more
Secondary outcome measures
CGD-related IBD: 1.Change in modified HBI. 2.Change in histopathological endoscopy.
Gene expression: 1.Change in IFN gene module enrichment score derived from whole blood RNA expression data.
Inflammatory lung disease: 1. Change in FEV1. 2. Change in DLCO. 3.Change in CT radiography. 4. Change in 6-minute walk.
+1 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: XELJANZ (tofacitinib)Experimental Treatment1 Intervention
Tofacitinib is self-administered orally at 5 mg twice per day or 11 mg once per day for 3 months.

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,268 Previous Clinical Trials
5,480,693 Total Patients Enrolled
Christa S Zerbe, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
7 Previous Clinical Trials
592 Total Patients Enrolled

Media Library

Tofacitinib (Janus Kinase (JAK) Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05104723 — Phase 1 & 2
Chronic Granulomatous Disease Research Study Groups: XELJANZ (tofacitinib)
Chronic Granulomatous Disease Clinical Trial 2023: Tofacitinib Highlights & Side Effects. Trial Name: NCT05104723 — Phase 1 & 2
Tofacitinib (Janus Kinase (JAK) Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05104723 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are people still able to sign up for this clinical trial?

"This clinical trial is not accepting patients at the moment. The listing was originally published on August 12th, 2022 and was last updated September 3rd, 2022. 438 other trials are actively recruiting patients with hepatobiliary disorders while 34 studies for XELJANZ (tofacitinib) currently have open enrolment."

Answered by AI

What is the main goal of this research project?

"The primary objective of this study, which will run from Day 1 to Day 120, is to evaluate the rate of treatment-related toxicities. Additionally, the investigators hope to assess the change in modified HBI scores, histopathological endoscopy changes, gene expression IFN-induced module enrichment score, and skin flares or ulcerations captured by photography."

Answered by AI

What are the main purposes that XELJANZ (tofacitinib) is used for?

"XELJANZ (tofacitinib) is most commonly used to treat juvenile arthritis, though it can also help patients who are intolerant to methotrexate or have had an inadequate response to other forms of therapy."

Answered by AI

Is XELJANZ (tofacitinib) a new medication?

"At this moment, there are 34 on-going clinical trials assessing the efficacy of XELJANZ (tofacitinib). Of these research projects, 7 have reached Phase 3. While a number of the experiments are based in Shanghai, China; there are 662 locations worldwide where XELJANZ (tofacitinib) is being studied."

Answered by AI

How many people have volunteered to take part in this experiment?

"As of right now, this trial is not searching for new participants. However, this could change in the future as the clinical trial was only most recently edited on September 3rd, 2022. For other potential studies, 438 trials are currently enrolling patients with hepatobiliary disorders and 34 XELJANZ (tofacitinib) trials have open enrollment."

Answered by AI
~10 spots leftby Jan 2026