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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Study ID
NCT05104723
Phase
PHASE1/PHASE2
Status
Completed

Conditions

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

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • XELJANZ (tofacitinib) — DRUG
    The XELJANZ 5-mg tablets or 11-mg XR tablets will taken orally twice daily or once daily, respectively, in this study.

Study Details

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....

Key Dates

Start date
Aug 12, 2022
Status verified
May 2026
Primary completion
May 4, 2026
Completion
May 4, 2026

Study Design

Enrollment
10 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: XELJANZ (tofacitinib)
    Tofacitinib is self-administered orally at 5 mg twice per day or 11 mg once per day for 3 months.

Primary Outcome Measure

Rate of treatment-related toxicities. [ Time Frame: Day 1 through Day 120 ]

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892-

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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