MAS825 for Autoinflammatory Diseases

(MASter-1 Trial)

Not currently recruiting at 37 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of a new drug, MAS825, for individuals with certain rare genetic conditions that cause immune system overactivity. These conditions include NLRC4-GOF, XIAP deficiency, or CDC42 mutations, which can lead to persistent inflammation and digestive issues. Participants will receive either MAS825 or a placebo (a dummy treatment) to compare outcomes. This trial suits those with one of these genetic diagnoses who continue to experience symptoms despite other treatments. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Studies have shown that MAS825 has been tested for safety in patients with severe COVID-19 pneumonia. While specific safety data for MAS825 in autoinflammatory diseases is lacking, similar treatments known as biologics have been used safely in other contexts. This suggests that MAS825 might be well-tolerated, though more evidence is still being gathered. As this trial is in Phase 2, the treatment has demonstrated some safety in earlier tests, but additional data is needed for confirmation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autoinflammatory diseases?

Researchers are excited about MAS825 because it targets autoinflammatory diseases differently from existing treatments. While most current options focus on general anti-inflammatory effects, MAS825 works by specifically inhibiting a protein called IL-1β, which plays a key role in the inflammation process. This targeted approach could lead to more effective symptom control with potentially fewer side effects. Additionally, MAS825 may offer quicker relief compared to traditional treatments, making it a promising option for those seeking faster improvements in their condition.

What evidence suggests that MAS825 might be an effective treatment for autoinflammatory diseases?

Research has shown that MAS825, a special type of antibody, holds promise for treating certain inflammatory diseases. In earlier studies, patients experienced improvements in breathing, exercise ability, and overall well-being after 10 months of treatment. Long-term use of MAS825 also benefited patients with systemic juvenile idiopathic arthritis (sJIA) and recurrent episodes of macrophage activation syndrome (MAS). This trial will compare MAS825 to a placebo to evaluate its effectiveness in reducing inflammation and related symptoms in individuals with genetic inflammatory conditions. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11879313/

Long-term efficacy of MAS825, a bispecific anti-IL1β and IL ... After 10 months of treatment, the patient presented an improvement in oxygen saturation, exercise tolerance and quality of life, allowing glucocorticoid ...

2. academic.oup.com academic.oup.com/rheumatology/advance-article/doi/10.1093/rheumatology/keae440/7739895

Long-term efficacy of MAS825, a bispecific anti-IL1β and IL-18 ... Long-term efficacy of MAS825, a bispecific anti-IL1β and IL-18 monoclonal antibody, in two patients with sJIA and recurrent episodes of MAS.

3. withpower.com withpower.com/trial/phase-3-enterocolitis-11-2020-3d26f

MAS825 for Autoinflammatory Diseases (MASter-1 Trial) Trial Overview The trial is testing the effectiveness of MAS825 compared to a placebo in treating autoinflammatory conditions caused by specific genetic ...

4. clinicaltrials.eu clinicaltrials.eu/inn/human-igg1-monoclonal-antibody-against-human-il-1-beta-and-human-il-18/

MAS825: A Promising Treatment for Autoinflammatory ... This innovative treatment is being studied for its efficacy in managing autoinflammatory diseases and reducing inflammation in patients with coronary heart ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39178016/

Long-term efficacy of MAS825, a bispecific anti-IL1β and IL ... Long-term efficacy of MAS825, a bispecific anti-IL1β and IL-18 monoclonal antibody, in two patients with systemic JIA and recurrent episodes of ...

6. ctv.veeva.com ctv.veeva.com/study/study-to-evaluate-the-efficacy-safety-and-tolerability-of-mas825-in-patients-with-monogenic-il-18-d

Study to Evaluate the Efficacy, Safety and Tolerability of ... This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, ...