M5049 for Myositis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test whether the oral drug M5049 (also known as Enpatoran) is effective and safe for individuals with certain types of muscle inflammation, specifically dermatomyositis (DM) and polymyositis (PM). Participants will receive either M5049 or a placebo (a pill with no active treatment) to determine if the drug can help manage their condition over 24 weeks. The trial seeks individuals with DM or PM who have active symptoms, such as muscle weakness or skin rash, despite being on standard treatment. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.
Will I have to stop taking my current medications?
The trial allows participants to continue stable doses of oral corticosteroids and one non-corticosteroid immunosuppressive medication. It does not specify if other medications need to be stopped, so you may need to discuss your specific medications with the trial team.
Is there any evidence suggesting that M5049 is likely to be safe for humans?
Research has shown that M5049, also known as enpatoran, has undergone safety testing in humans. A previous study with healthy participants found M5049 to be generally safe and well-tolerated, with mostly mild side effects reported.
The current study is in an early stage, where researchers are carefully assessing the safety of M5049 for individuals with dermatomyositis and polymyositis. This phase identifies potential side effects and their frequency, but it does not yet provide complete safety information.
M5049 is not yet approved for treating these specific conditions, making this study crucial for understanding its safety for future patients.12345Why do researchers think this study treatment might be promising for myositis?
Unlike the standard treatments for myositis, which often rely on immunosuppressants and corticosteroids, M5049 is unique because it targets specific pathways involved in the inflammatory process. Researchers are excited about M5049 because it works by inhibiting the Toll-like receptor (TLR) pathways, which play a critical role in the body's immune response. This targeted approach has the potential to reduce inflammation more effectively and with fewer side effects compared to broader immunosuppressive therapies. Additionally, M5049's focus on these specific pathways could offer a new hope for patients who do not respond well to existing treatments.
What evidence suggests that M5049 might be an effective treatment for myositis?
Research has shown that M5049, also known as enpatoran, could be a promising treatment for conditions like dermatomyositis (DM) and polymyositis (PM). It blocks certain proteins, TLR7 and TLR8, which play a role in the body's immune response. Early studies found enpatoran to be safe and tolerable, and ongoing research is exploring its full potential for treating inflammatory diseases. In this trial, participants will receive either a high dose of M5049 or a placebo. Although specific data on its effectiveness for DM and PM is still being gathered, its mechanism suggests it could help reduce inflammation and muscle damage in these conditions.23456
Who Is on the Research Team?
Medical Responsible
Principal Investigator
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Are You a Good Fit for This Trial?
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.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive orally administered M5049 or placebo for 24 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may opt into continuation of treatment with M5049 high dose long-term
What Are the Treatments Tested in This Trial?
Interventions
- M5049
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
EMD Serono Research & Development Institute, Inc.
Lead Sponsor
Miguel Fernández Alcalde
EMD Serono Research & Development Institute, Inc.
Chief Executive Officer
Bachelor’s Degree in Pharmacy from the University Complutense in Madrid, MBA from the University of Alcalá de Henares, Master’s Degree in Management from IESE Business School
Danny Bar-Zohar
EMD Serono Research & Development Institute, Inc.
Chief Medical Officer since 2022
MD
Merck KGaA, Darmstadt, Germany
Industry Sponsor
Danny Bar-Zohar
Merck KGaA, Darmstadt, Germany
Chief Medical Officer since 2022
MD
Belén Garijo
Merck KGaA, Darmstadt, Germany
Chief Executive Officer since 2021
MD