What side effects are associated with this type of intervention?

Common treatments for Polymyositis include intravenous immune globulin (IVIG), rituximab, and mycophenolate mofetil. IVIG is generally well-tolerated but can cause headaches and, rarely, more serious side effects like renal dysfunction. Rituximab can lead to infusion reactions, infections such as pneumonia and cellulitis, and other serious adverse effects like heart failure and respiratory issues. Mycophenolate mofetil may cause gastrointestinal distress, increased risk of infections, and, in some cases, serious opportunistic infections. These treatments, while effective, require careful monitoring for adverse effects.

What prior FDA approvals exist?

There are no specific mentions of FDA-approved treatments for Polymyositis that are similar to the TLR7 and TLR8 inhibitor M5049 in the provided context. However, treatments for Polymyositis often involve immunosuppressive agents such as methotrexate, azathioprine, and mycophenolate mofetil, which are used to manage the immune response. These treatments aim to reduce inflammation and improve muscle strength in patients with Polymyositis.

What other types of research exist?

Promising novel alternatives to M5049 for Polymyositis patients include: <ol> <li>Intravenous immunoglobulin (IVIG) - Shown to be effective in improving muscle strength and function in PM patients.</li> <li></li> </ol> Janus kinase (JAK) inhibitors - Emerging as potential treatments due to their immunomodulatory effects. 3. Rituximab - A monoclonal antibody targeting CD20-positive B cells, showing promise in refractory cases of PM. 4. Abatacept - A T-cell co-stimulation modulator that has shown efficacy in some inflammatory myopathies. 5. Tocilizumab - An IL-6 receptor antagonist that may benefit patients with PM by reducing inflammation.

← Back to Search

Unknown

M5049 for Myositis

Q: What is the mechanism of action of this treatment?

Common treatments for Polymyositis include corticosteroids, immunosuppressants, and biologics. These treatments primarily work by reducing inflammation and modulating the immune system. Corticosteroids decrease inflammation by inhibiting multiple inflammatory pathways. Immunosuppressants, such as methotrexate and azathioprine, reduce immune system activity to prevent further muscle damage. Biologics, like rituximab, target specific components of the immune system to reduce inflammation. The M5049 trial focuses on TLR7 and TLR8 inhibitors, which block Toll-like receptor pathways involved in immune activation and inflammation. This is significant for Polymyositis patients as it offers a targeted approach to reduce muscle inflammation and improve muscle function, potentially with fewer side effects compared to broader immunosuppressive therapies.

Phase 2

Recruiting

Research Sponsored by EMD Serono Research & Development Institute, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Must not have

Primary diagnosis of juvenile DM, or adult participants previously diagnosed with juvenile DM

Severe interstitial lung disease defined as supplemental oxygen required at rest, or forced vital capacity (FVC) of <60 percent (%) predicted. Participants within 1 year of PM/DM diagnosis and anti-MDA5 antibody, should have been evaluated for interstitial lung disease (ILD) with high resolution computed tomography (HRCT) Chest

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing a pill called M5049 for people with muscle diseases known as dermatomyositis (DM) and polymyositis (PM). These conditions cause muscle inflammation and weakness. The pill aims to reduce inflammation and improve muscle strength over several months.

Who is the study for?

This trial is for adults with moderate to severe dermatomyositis (DM) or polymyositis (PM), who have certain levels of muscle weakness, skin rash, and elevated muscle enzymes. They must be on stable medication regimens and not have other conditions like juvenile DM, inclusion body myositis, cancer-associated myositis, or severe lung disease.

What is being tested?

The NEPTUNIA study tests the safety and effectiveness of a new oral drug called M5049 in treating inflammatory muscle diseases DM and PM over 24 weeks. Participants will either receive a high dose of M5049 or a placebo to compare outcomes.

What are the potential side effects?

While specific side effects are not listed here, common risks may include digestive issues, allergic reactions, fatigue, liver problems due to medication intake. The exact side effects will be monitored throughout the trial.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I was diagnosed with juvenile dermatomyositis.

Select...

I need oxygen or have severe lung issues, and I've been checked for lung disease with a special scan.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Open Label Extension (OLE) Period: M5049 high doseExperimental Treatment1 Intervention

Group II: Double-blind Placebo Controlled (DBPC) Period: M5049 high doseExperimental Treatment1 Intervention

Group III: DBPC Period: PlaceboPlacebo Group1 Intervention

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Polymyositis include corticosteroids, immunosuppressants, and biologics. These treatments primarily work by reducing inflammation and modulating the immune system. Corticosteroids decrease inflammation by inhibiting multiple inflammatory pathways. Immunosuppressants, such as methotrexate and azathioprine, reduce immune system activity to prevent further muscle damage. Biologics, like rituximab, target specific components of the immune system to reduce inflammation. The M5049 trial focuses on TLR7 and TLR8 inhibitors, which block Toll-like receptor pathways involved in immune activation and inflammation. This is significant for Polymyositis patients as it offers a targeted approach to reduce muscle inflammation and improve muscle function, potentially with fewer side effects compared to broader immunosuppressive therapies.

P2X7 Receptor Antagonist Reduces Fibrosis and Inflammation in a Mouse Model of Alpha-Sarcoglycan Muscular Dystrophy.Activation of TLR4 induces inflammatory muscle injury via mTOR and NF-κB pathways in experimental autoimmune myositis mice.GSI Treatment Preserves Protein Synthesis in C2C12 Myotubes.