M5049 for Myositis

Recruiting at 33 trial locations

Overseen By Communication Center

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: EMD Serono Research & Development Institute, Inc.

Must be taking: Corticosteroids, Immunosuppressives

Disqualifiers: Inclusion body myositis, Cancer, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

40 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

Research Team

Medical Responsible

Principal Investigator

Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

Eligibility Criteria

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

I have been diagnosed with dermatomyositis or polymyositis and have positive autoantibodies.

I have moderate to severe muscle weakness and at least two other symptoms of myopathy.

I have active myositis or a skin rash from myositis, confirmed by tests in the last 6 months.

Exclusion Criteria

I do not have any uncontrolled health conditions that could risk my participation in the study.

I was diagnosed with juvenile dermatomyositis.

I have been diagnosed with a specific muscle disease and screened for cancer as recommended.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive orally administered M5049 or placebo for 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of treatment with M5049 high dose long-term

Treatment Details

Interventions

• M5049
• Placebo

Trial Overview 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.

Participant Groups

Group I: Open Label Extension (OLE) Period: M5049 high dose Experimental Treatment 1 Intervention

Group II: Double-blind Placebo Controlled (DBPC) Period: M5049 high dose Experimental Treatment 1 Intervention

Group III: DBPC Period: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

EMD Serono Research & Development Institute, Inc.

Lead Sponsor

Trials

86

Recruited

22,700+

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

Trials

449

Recruited

122,000+

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