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)

Status: Terminated
Location: See all (30) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:

• 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

Locations
United States
Arizona
Neuromuscular Research Center
Phoenix
HonorHealth Research Institute - Bob Bove Neuroscience Institute-Neuroscience Research
Scottsdale
Mayo Clinic Scottsdale (6365)
Scottsdale
Colorado
Barbara Davis Center
Aurora
Florida
HMD Research LLC
Orlando
Bolanos Clinical Research
Pembroke Pines
Georgia
Augusta University-Rheumatology
Augusta
Maryland
Johns Hopkins University - Department of Medicine, Division of Rheumatology
Baltimore
Minnesota
University of Minnesota-Dermatology
Minneapolis
Missouri
University of Kansas Medical Center-Neuromuscular
Kansas City
Pennsylvania
University of Pittsburgh
Pittsburgh
Texas
Austin Neuromuscular Center
Austin
Nerve and Muscle Center of Texas-Clinical research
Houston
Other Locations
Greece
Hippokration Hospital - 2nd Department of Medicine and Laboratory
Athens
National and Kapodistrian University of Athens (Egnitio Hospital)
Athens
University General Hospital of Larissa
Larissa
Italy
Azienda Ospedaliero Universitaria Policlinico G. Rodolico-San Marco Di Catania (Vittorio Emanuele) - Reumatologia
Catania
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania
Catania
Azienda Usl Toscana Centro
Florence
Arcispedale S. Maria Nuova
Reggio Emilia
Fondazione Policlinico Universitario A. Gemelli-IRCCS, UCSC - Scienze Mediche e Chirurgiche
Rome
Poland
Instytut Reumatologii im. Eleonory Reicher - Department of Connective Tissue Diseases
Warsaw
Spain
CHUAC - Complexo Hospitalario Universitario A Coruña - Rheumatology
A Coruña
Hospital Vall d'Hebron
Barcelona
Hospital Universitario Ramon y Cajal, Madrid - Rheumatology Department
Madrid
United Kingdom
Doncaster Royal Infirmary (3466)
Doncaster
Royal Free London NHS Foundation Trust
London
University College London Hospitals NHS Foundation Trust- Neuromuscular Diseases
London
Salford Royal Hospital, Barnes Clinical Research Facility
Salford
Royal Wolverhampton Hospitals (6493)
Wolverhampton
Time Frame
Start Date: 2023-01-19
Completion Date: 2025-06-25
Participants
Target number of participants: 40
Treatments
Experimental: Double-blind Placebo Controlled (DBPC) Period: M5049 high dose
Placebo_comparator: DBPC Period: Placebo
Experimental: Open Label Extension (OLE) Period: M5049 high dose
Related Therapeutic Areas
Sponsors
Collaborators: Merck KGaA, Darmstadt, Germany
Leads: EMD Serono Research & Development Institute, Inc.

This content was sourced from clinicaltrials.gov