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

Must not be taking: Anti-rejection, Immunomodulatory

Disqualifiers: HIV, Hepatitis B/C, Tuberculosis, others

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.

Do I have to stop taking my current medications for the MAS825 trial?

The trial requires that you stop taking certain medications, specifically anti-rejection and immunomodulatory drugs, within 28 days or 5 half-lives before starting MAS825, except for glucocorticoids, cyclosporin, and targeted therapies. The protocol does not specify other medications, so it's best to discuss with the trial team.

What data supports the effectiveness of the drug MAS825 for autoinflammatory diseases?

Research indicates that treatments targeting specific immune responses, like anti-IL1α and β-blocking agents, have significantly improved outcomes in autoinflammatory disorders, suggesting that similar approaches, such as MAS825, may also be effective. ^{1 2 3 4 5}

What safety data exists for MAS825 in treating autoinflammatory diseases?

The research does not provide specific safety data for MAS825, but it mentions that biologics, which are similar types of treatments, have been used safely in autoinflammatory diseases. These treatments have improved outcomes, although challenges remain in drug therapy for these conditions. ^{1 3 6 7 8}

How is the drug MAS825 different from other treatments for autoinflammatory diseases?

MAS825 is unique because it targets specific inflammatory pathways involved in autoinflammatory diseases, potentially offering a novel approach compared to existing treatments. While other treatments may focus on general inflammation, MAS825's mechanism may involve targeting specific cytokines like IL-18, which are key in these conditions. ^{1 2 5 9 10}

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Period 1: Open-label Active Treatment

Participants receive MAS825 in an open-label, single-arm active treatment

Approximately 1 year

Period 2: Randomized-withdrawal, Double-blind, Placebo-controlled

Participants are randomized to receive either MAS825 or placebo to evaluate the prevention of disease flares

Approximately 1 year

Period 3: Open-label Long-term Safety Follow-up

Participants enter an open-label phase for long-term safety follow-up

Approximately 1-2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

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

References

1. Germany pubmed.ncbi.nlm.nih.gov

[Efficacy and safety analysis of off-label treatment with biologics in autoinflammatory diseases : Experiences from a German registry (GRAID2)]. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Development and preselection of criteria for short term improvement after anti-TNF alpha treatment in ankylosing spondylitis. [2022]

3. Germany pubmed.ncbi.nlm.nih.gov

Regulating against the dysregulation: new treatment options in autoinflammation. [2018]

4. England pubmed.ncbi.nlm.nih.gov

Double blind, randomised, placebo controlled study of leflunomide in the treatment of active ankylosing spondylitis. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Outcomes of a multicentre randomised clinical trial of etanercept to treat ankylosing spondylitis. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Open label trial of anakinra in active ankylosing spondylitis over 24 weeks. [2008]

8. Switzerland pubmed.ncbi.nlm.nih.gov

The Autoinflammatory Diseases Alliance Registry of monogenic autoinflammatory diseases. [2022]

9. Switzerland pubmed.ncbi.nlm.nih.gov

IL-18 in Autoinflammatory Diseases: Focus on Adult Onset Still Disease and Macrophages Activation Syndrome. [2023]

10. Switzerland pubmed.ncbi.nlm.nih.gov

OLT1177 (Dapansutrile), a Selective NLRP3 Inflammasome Inhibitor, Ameliorates Experimental Autoimmune Encephalomyelitis Pathogenesis. [2020]