MAS825 for Autoinflammatory Diseases
(MASter-1 Trial)

Recruiting in Palo Alto (17 mi)

+28 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Novartis Pharmaceuticals

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This trial is testing a new medication called MAS825 in patients with rare genetic conditions that don't respond well to typical treatments. The study will take several years to evaluate the medication's effectiveness and safety. The goal is to see if MAS825 can help manage these conditions by targeting the underlying genetic problems.

Eligibility Criteria

This trial is for patients with specific genetic autoinflammatory diseases (NLRC4-GOF, XIAP deficiency, or CDC42 mutations) who weigh at least 3 kg. Participants must provide informed consent and have active disease symptoms. Excluded are those with hypersensitivity to study drugs, positive HIV/Hepatitis tests, tuberculosis infection, certain CDC42 mutation syndromes, recent anti-rejection/immunomodulatory drug use except some exceptions like glucocorticoids.

Inclusion Criteria

I weigh at least 3 kg.

I have NLRC4-GOF, XIAP deficiency, or CDC42 mutations and am in a Novartis MAP for MAS825.

I have a genetic condition related to NLRC4, XIAP, or CDC42 and show signs of inflammation.

See 1 more

Exclusion Criteria

Pregnant or nursing (lactating) females.

I agree to use effective birth control during MAS825 therapy.

I tested positive for tuberculosis recently.

See 10 more

Treatment Details

Interventions

MAS825 (Other)
Placebo (Other)

Trial OverviewThe trial is testing the effectiveness of MAS825 compared to a placebo in treating autoinflammatory conditions caused by specific genetic mutations. It's a Phase 2 study focusing on safety and how well patients tolerate the treatment.

Participant Groups

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MAS825Experimental Treatment1 Intervention

Experimental drug

Group II: PlaceboPlacebo Group1 Intervention

matching placebo