Study of M5049 in DM and PM Participants (NEPTUNIA)

NCT ID: NCT05650567

Last Updated: 2025-07-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-01-19

Study Completion Date

2025-06-25

Brief Summary

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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.

Detailed Description

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Conditions

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Dermatomyositis Polymyositis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Double-blind Placebo Controlled (DBPC) Period: M5049 high dose

Group Type EXPERIMENTAL

M5049 high dose

Intervention Type DRUG

Participants will receive film-coated tablets of M5049 at a high dose orally, twice daily up to 24 weeks.

DBPC Period: Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants will receive placebo matched to M5049 orally, twice daily up to 24 weeks.

Open Label Extension (OLE) Period: M5049 high dose

Group Type EXPERIMENTAL

M5049 high dose

Intervention Type DRUG

Participants will receive film-coated tablets of M5049 at a high dose orally, twice daily up to 24 weeks.

Interventions

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M5049 high dose

Participants will receive film-coated tablets of M5049 at a high dose orally, twice daily up to 24 weeks.

Intervention Type DRUG

Placebo

Participants will receive placebo matched to M5049 orally, twice daily up to 24 weeks.

Intervention Type DRUG

Other Intervention Names

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Enpatoran

Eligibility Criteria

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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)

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Merck KGaA, Darmstadt, Germany

INDUSTRY

Sponsor Role collaborator

EMD Serono Research & Development Institute, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Responsible

Role: STUDY_DIRECTOR

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

Locations

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Neuromuscular Research Center

Phoenix, Arizona, United States

Site Status

HonorHealth Research Institute - Bob Bove Neuroscience Institute-Neuroscience Research

Scottsdale, Arizona, United States

Site Status

Mayo Clinic Scottsdale (6365)

Scottsdale, Arizona, United States

Site Status

Barbara Davis Center

Aurora, Colorado, United States

Site Status

HMD Research LLC

Orlando, Florida, United States

Site Status

Bolanos Clinical Research

Pembroke Pines, Florida, United States

Site Status

Augusta University-Rheumatology

Augusta, Georgia, United States

Site Status

Johns Hopkins University - Department of Medicine, Division of Rheumatology

Baltimore, Maryland, United States

Site Status

University of Minnesota-Dermatology

Minneapolis, Minnesota, United States

Site Status

University of Kansas Medical Center-Neuromuscular

Kansas City, Missouri, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Austin Neuromuscular Center

Austin, Texas, United States

Site Status

Nerve and Muscle Center of Texas-Clinical research

Houston, Texas, United States

Site Status

Institute of Rheumatology - Rheumatology

Prague, , Czechia

Site Status

Hippokration Hospital - 2nd Department of Medicine and Laboratory

Athens, , Greece

Site Status

National and Kapodistrian University of Athens (Egnitio Hospital)

Athens, , Greece

Site Status

University General Hospital of Larissa

Larissa, , Greece

Site Status

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

Catania, , Italy

Site Status

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

Catania, , Italy

Site Status

Azienda Usl Toscana Centro

Florence, , Italy

Site Status

Arcispedale S. Maria Nuova

Reggio Emilia, , Italy

Site Status

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

Rome, , Italy

Site Status

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

Warsaw, , Poland

Site Status

CHUAC - Complexo Hospitalario Universitario A Coruña - Rheumatology

A Coruña, , Spain

Site Status

Hospital Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital Universitario Ramon y Cajal, Madrid - Rheumatology Department

Madrid, , Spain

Site Status

Doncaster Royal Infirmary (3466)

Doncaster, , United Kingdom

Site Status

Royal Free London NHS Foundation Trust

London, , United Kingdom

Site Status

University College London Hospitals NHS Foundation Trust- Neuromuscular Diseases

London, , United Kingdom

Site Status

Salford Royal Hospital, Barnes Clinical Research Facility

Salford, , United Kingdom

Site Status

Royal Wolverhampton Hospitals (6493)

Wolverhampton, , United Kingdom

Site Status

Countries

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United States Czechia Greece Italy Poland Spain United Kingdom

Related Links

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https://medical.emdserono.com/en_US/home.html

US Medical Information website, Medical Resources

Other Identifiers

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2022-501351-82-00

Identifier Type: OTHER

Identifier Source: secondary_id

MS200569_0041

Identifier Type: -

Identifier Source: org_study_id

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