Phase IIa Study of Enpatoran in DM and PM
Research type
Research Study
Full title
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)
IRAS ID
1006534
Contact name
Communication Center Merck KGaA Communication Center Merck KGaA
Contact email
Sponsor organisation
Merck Healthcare KGaA, Darmstadt, Germany
Clinicaltrials.gov Identifier
Research summary
Enpatoran (formerly M5049) is a novel orally administered small molecule dual toll-like receptor 7 and 8 (TLR7 and TLR8) antagonist (a class of proteins that play a key role in the non-specific immune system response) developed by Merck Healthcare KGaA for the treatment of immunological disorders (dysfunction of the immune system) like systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE), Dermatomyositis (DM) and Polymyositis (PM).
This study intends to evaluate the efficacy and safety of orally administered enpatoran in idiopathic inflammatory myopathies (IIMs), specifically DM and PM patients in a randomized, parallel, double-blind, placebo controlled (DBPC) 24 week study. IIMs are a group of chronic autoimmune diseases in which inflammation occurs in muscles and often in organs and tissues other than muscle, including the skin in DM. This study offers an optional 24-week open-label extension (OLE) treatment period for completers of the 24 week double-blind treatment period. This study will significantly contribute to further understanding of the immunopathogenesis (development of disease as affected by the immune system) of DM and PM.
Forty (40) adult participants with active DM or PM with moderate to severe myopathy (disease of muscle tissue) will be enrolled to receive enpatoran - at study dose twice daily or placebo stratified according to disease subtype per 2017 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria (that provides a patient's probability of having IIM for use in clinical and research settings) at approximately 29 study centers in the UK, Unites States, and Europe.REC name
South Central - Berkshire B Research Ethics Committee
REC reference
22/SC/0418
Date of REC Opinion
20 Dec 2022
REC opinion
Further Information Favourable Opinion