17 Participants Needed

MAS825 for Autoinflammatory Diseases

(MASter-1 Trial)

Recruiting at 28 trial locations
NP
Overseen ByNovartis Pharmaceuticals
Age: Any Age
Sex: Any
Trial Phase: Phase 2
Sponsor: Novartis Pharmaceuticals
Prior Safety DataThis treatment has passed at least one previous human trial

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
  • Placebo
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

Find a Clinic Near You

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 profile image

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye profile image

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD