Tofacitinib for Chronic Granulomatous Disease

Overseen by Christa S Zerbe, M.D.

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing tofacitinib, a pill that reduces inflammation, in adults with chronic granulomatous disease (CGD) who haven't responded to other treatments. The drug aims to calm the overactive immune system in these patients. Tofacitinib has been approved for the treatment of rheumatoid arthritis, psoriatic arthritis, and ulcerative colitis.

Research Team

Christa S Zerbe, M.D.

Principal Investigator

National Institute of Allergy and Infectious Diseases (NIAID)

Eligibility Criteria

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.

Inclusion Criteria

Participants who can become pregnant or impregnate their partner must agree to use at least one highly effective method of contraception

I am 18 years old or older.

I have IBD, lung, or skin disease related to CGD diagnosed through endoscopy, imaging, or PFTs.

Exclusion Criteria

Your blood tests show very high levels of certain enzymes, or very low levels of white blood cells.

I do not have any health conditions that could affect the study's treatment evaluation.

I currently use inhaled tobacco, vaping products, cannabis, or other illicit drugs.

Treatment Details

Interventions

• Tofacitinib (Janus Kinase (JAK) Inhibitor)

Trial Overview 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.

Participant Groups

Group I: XELJANZ (tofacitinib) Experimental Treatment 1 Intervention

Tofacitinib is self-administered orally at 5 mg twice per day or 11 mg once per day for 3 months.

Who Is Running the Clinical Trial?

National Institute of Allergy and Infectious Diseases (NIAID)

Lead Sponsor

Trials

3,361

Recruited

5,516,000+

Dr. Jeanne Marrazzo

National Institute of Allergy and Infectious Diseases (NIAID)

Chief Executive Officer since 2023

MD, MPH

Dr. H. Clifford Lane

National Institute of Allergy and Infectious Diseases (NIAID)

Chief Medical Officer

MD