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A phase IIa, randomized, parallel, Double-Blind, placebo controlled study to evaluate the efficacy and safety of enpatoran in dermatomyositis and polymyositis participants receiving standard of care (NEPTUNIA)

NCT ID NCT05650567

NEW Sponsor: EMD Serono Research & Development Institute, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 09, 2026 · Last updated Sep 09, 2026

Summary

The purpose of this study is to evaluate the efficacy and safety of orally administered M5049 in idiopathic inflammatory myopathies, specifically dermatomyositis (DM) and polymyositis (PM) participants for 24 weeks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

20 people

The number who actually took part.

Started

Jan 2023

Finished

Jun 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of probable or definite DM or PM as per 2017 ACR/EULAR classification criteria, with positive autoantibody status. Anti-synthetase syndrome (ASyS) participants that meet classification criteria are allowed * Active disease on standard of care (SoC), must meet 1 of the criteria within 6 months prior to Screening: Pathological evidence of active myositis in muscle biopsy; Evidence of active myositis by Electromyography (EMG); Magnetic resonance imaging (MRI) with evidence of active myositis; or any muscle enzyme greater than or equal to (\>=) 4 × upper limit of normal (ULN) at time of Screening; Active PM/DM skin rash as per cutaneous dermatomyositis area and severity index-A (CDASI-A) \>= 7 at time of Screening * Minimum disease severity defined by: moderate to severe myopathy with manual muscle testing-8 (MMT-8) \>= 80 and less than or equal to (\<=) 142 AND at least 2 of the following core set measures (CSM) abnormalities: Patient Global Activity (PtGA) \>= 2 centimeters (cm); Physician Global Activity (PGA) derived from myositis disease activity assessment tool (MDAAT) \>= 2 cm; Extramuscular Activity Assessment derived from MDAAT \>2 cm; At least 1 muscle enzyme \> 1.5 times ULN; health assessment questionnaire-disability index (HAQ-DI) \>= 0.25 * Stable doses of oral corticosteroids (CS) and/or maximum of 1 non-corticosteroid immunosuppressive/immunomodulatory medications (methotrexate, 6 mercaptopurine, sulfasalazine, mycophenolate mofetil or sodium, azathioprine, leflunomide, cyclosporine, oral tacrolimus) for DM or PM * Participants have a body mass index (BMI) lower or Equal to 40.0 kilograms per square meter (kg/m\^2) * Other protocol defined inclusion criteria could apply Exclusion Criteria: * Primary diagnosis of inclusion body myositis (IBM), malignancy-associated myositis (defined as diagnosis of myositis within 3 years of cancer), immune mediated necrotizing myopathy (IMNM) with a biopsy characterized as necrotizing biopsy or IMNM with positive anti-signal recognition particle antibody (SRP) or anti 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) auto antibodies. Participants with anti-transcription intermediary factor 1 (TIF1) gamma antibody or newly diagnosed (within 1 year) anti MDAT5 antibody should have had adequate screening for cancer within 12 months of Day 1. Adequate screening of cancer is defined as up-to-date age and gender appropriate screening as per national guidelines * Primary diagnosis of juvenile DM, or adult participants previously diagnosed with juvenile DM * Any other active concurrent connective tissue disease associated with inflammatory myopathy in the Investigator's opinion. Eligibility of participants with diagnosis of concurrent connective tissue disease(s) will be reviewed and approved by an idiopathic inflammatory myopathies (IIM) expert committee * 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 * Any uncontrolled disease (for example \[e.g.\], severe respiratory, cardiovascular, gastrointestinal, neurological, psychiatric, hematological, metabolic \[including thyroiditis with increased/decreased thyroid stimulating hormone (TSH)\], renal \[Estimated glomerular filtration rate \< 40 milliliter per minute/1.73 m\^2 as calculated by the Modification of Diet in Renal Disease equation by the central laboratory\], hepatic, endocrine/reproductive organ disease) other than DM/PM, that in the Investigator's or Sponsor/designee's opinion constitutes an inappropriate risk or contraindication for participation in the study or that could interfere with the study objectives, conduct, or evaluation * Other protocol defined exclusion criteria could apply

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Conditions

The condition(s) this trial relates to.

dermatomyositis myositis disease polymyositis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Dermatomyositis Polymyositis

Contacts and locations

Locations

  • Arcispedale S. Maria Nuova

    Reggio Emilia, Italy

  • Augusta University-Rheumatology

    Augusta, Georgia, 30912, United States

  • Austin Neuromuscular Center

    Austin, Texas, 78759, United States

  • Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco Di Catania (Vittorio Emanuele) - Reumatologia

    Catania, Italy

  • Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania

    Catania, Italy

  • Azienda Usl Toscana Centro

    Florence, Italy

  • Barbara Davis Center

    Aurora, Colorado, 80045, United States

  • Bolanos Clinical Research

    Pembroke Pines, Florida, 33026, United States

  • CHUAC - Complexo Hospitalario Universitario A Coruña - Rheumatology

    A Coruña, Spain

  • Doncaster Royal Infirmary (3466)

    Doncaster, United Kingdom

  • Fondazione Policlinico Universitario A. Gemelli-IRCCS, UCSC - Scienze Mediche e Chirurgiche

    Rome, Italy

  • HMD Research LLC

    Orlando, Florida, 32819, United States

  • Hippokration Hospital - 2nd Department of Medicine and Laboratory

    Athens, Greece

  • HonorHealth Research Institute - Bob Bove Neuroscience Institute-Neuroscience Research

    Scottsdale, Arizona, 85251, United States

  • Hospital Universitario Ramon y Cajal, Madrid - Rheumatology Department

    Madrid, Spain

  • Hospital Vall d'Hebron

    Barcelona, Spain

  • Institute of Rheumatology - Rheumatology

    Prague, Czechia

  • Instytut Reumatologii im. Eleonory Reicher - Department of Connective Tissue Diseases

    Warsaw, Poland

  • Johns Hopkins University - Department of Medicine, Division of Rheumatology

    Baltimore, Maryland, 21224, United States

  • Mayo Clinic Scottsdale (6365)

    Scottsdale, Arizona, 85259, United States

  • National and Kapodistrian University of Athens (Egnitio Hospital)

    Athens, Greece

  • Nerve and Muscle Center of Texas-Clinical research

    Houston, Texas, 77030, United States

  • Neuromuscular Research Center

    Phoenix, Arizona, 85028, United States

  • Royal Free London NHS Foundation Trust

    London, United Kingdom

  • Royal Wolverhampton Hospitals (6493)

    Wolverhampton, United Kingdom

  • Salford Royal Hospital, Barnes Clinical Research Facility

    Salford, United Kingdom

  • University College London Hospitals NHS Foundation Trust- Neuromuscular Diseases

    London, United Kingdom

  • University General Hospital of Larissa

    Larissa, Greece

  • University of Kansas Medical Center-Neuromuscular

    Kansas City, Missouri, 66103, United States

  • University of Minnesota-Dermatology

    Minneapolis, Minnesota, 55455, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

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