MAS825 for Autoinflammatory Diseases

(MASter-1 Trial)

Recruiting at 28 trial locations

Overseen By Novartis Pharmaceuticals

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

17 Participants Needed

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.

Exclusion Criteria

Pregnant or nursing (lactating) females.

I agree to use effective birth control during MAS825 therapy.

I tested positive for tuberculosis recently.

Treatment Details

Interventions

• MAS825
• Placebo

Trial Overview The 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

Group I: MAS825 Experimental Treatment 1 Intervention

Experimental drug

Group II: Placebo Placebo Group 1 Intervention

matching placebo

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD