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Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications

NCT05104723 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-05-07

No results posted yet for this study

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....

Conditions

  • Chronic Granulomatous Disease
  • Inflammatory Gastrointestinal Disease
  • Inflammatory Skin Disease
  • Inflammatory Lung Disease

Interventions

DRUG

XELJANZ (tofacitinib)

The XELJANZ 5-mg tablets or 11-mg XR tablets will taken orally twice daily or once daily, respectively, in this study.

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Christa S Zerbe, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-12
Primary Completion
2026-05-04
Completion
2026-05-04
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05104723 on ClinicalTrials.gov