A Study to Understand How Nerandomilast Works in People With Active Idiopathic Inflammatory Myopathies

NCT07789015 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: 1305-0146U1111-1338-24542026-526365-24-00
• Study Type: interventional
• Target: 270
• Locations: 23 countries
• Sites: 138

Brief Summary

This study is open to adults aged 18 and older who have a condition known as active idiopathic inflammatory myopathy (IIM) or myositis. People can participate if they have a specific type of myositis. The purpose of this study is to find out whether a medicine called nerandomilast improves IIM symptoms. Participants are put into 2 groups randomly, which means by chance. One group takes nerandomilast tablets and the other group takes placebo tablets. Placebo tablets look like nerandomilast tablets but do not contain any medicine. Participants take nerandomilast or placebo twice a day for 1 year. Participants are in the study for about 1 year and 2 months. During this time, they visit the study site at least 16 times. During this time, doctors regularly check the participant's health, IIM symptoms, and take note of any unwanted effects. Participants fill in questionnaires about their IIM symptoms and how IIM impacts their quality of life. The results are compared between the 2 groups to see whether the treatment works.

Conditions

Idiopathic Inflammatory Myopathies

Outcome Measures

Primary Outcomes (1)

• Total improvement score (TIS) score (continuous) at Week 52

  The TIS composite score includes 6 core set measures: manual muscle testing (MMT8), extra-muscular disease activity (MDAAT), patient global disease activity (PtGA), physician global disease activity (PhGA), health assessment questionnaire disability index (HAQ-DI), and muscle enzymes. An improvement score for each core set measure will be assigned using the absolute percent change, based on predetermined ranges. The TIS will be the sum of the all core set measure, scoring from 0 to 100, with higher scores corresponding to a greater degree of improvement.

  At Week 52.

Secondary Outcomes (12)

• Achievement of a TIS-40 response (yes/no; defined as TIS ≥40) at Week 52

  At Week 52.

• Achievement of a successful tapering of oral corticosteroids (OCS) (yes/no) at Week 52

  At Week 52.

• Change from baseline in cutaneous dermatomyositis disease area and severity index activity score (CDASI-A) at Week 52 (in participants with baseline CDASI-A ≥6)

  At baseline and at Week 52.

• Change from baseline in extra-muscular disease activity as per myositis disease activity assessment tool (MDAAT) at Week 52

  At baseline and at Week 52.

• Change from baseline in PtGA at Week 52

  At baseline and at Week 52.

Study Arms (2)

Nerandomilast

EXPERIMENTAL

Drug: Nerandomilast

Placebo

PLACEBO COMPARATOR

Placebo matching nerandomilast

Drug: Placebo

Interventions

Placebo DRUG

Placebo-matching nerandomilast

Placebo

Nerandomilast DRUG

Nerandomilast

Nerandomilast

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female adult individuals from ≥18 years of age (or alternative age for adults based on local regulations) on the date of signing the informed consent.
• A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM) according to the 2017 American college of rheumatology (ACR)/European alliance of associations of rheumatology (EULAR) classification criteria.
• Participants with overlap myositis (OM) must have IIM as the predominant disease.
• Participants ≥18 years of age (or alternative age for adults based on local regulations) with juvenile dermatomyositis (JDM) can only be included if they were 15 years old or older at first onset of DM symptoms.
• For participants diagnosed with DM
• Disease activity defined by moderate to severe myopathy with manual muscle testing-8 (MMT-8) of ≥80 and ≤142/150 and cutaneous dermatomyositis disease area and severity index activity score (CDASI-A) ≥6 at screening and baseline and at least 2 of the following core set measures (CSM) abnormalities at screening:
• Patient global assessment (PtGA) ≥2 cm
• Physician global activity (PhGA) derived from myositis disease activity assessment tool (MDAAT) ≥2 cm
• Extramuscular disease activity as per MDAAT ≥2 cm
• At least 1 muscle enzyme (aldolase, creatine kinase (CK), aspartate aminotransferase (AST), alanine aminotransferase (ALT), or lactate dehydrogenase (LDH)) ≥1.5× upper limit of normal (ULN),
• Health assessment questionnaire-disability index (HAQ-DI) ≥0.25.
• For participants diagnosed with anti-synthetase syndrome (ASyS)\* and OM\*:
• Active disease based on at least 1 of the following criteria:
• CK ≥4× ULN at screening
• Muscle biopsy indicative of active IIM within up to 6 months prior to screening

You may not qualify if:

• Major surgery (major according to the investigator's assessment, e.g. hip replacement) performed within 6 weeks prior to randomisation or planned during the trial period.
• Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal cell carcinoma of the skin, or in situ squamous cell carcinoma of the skin, or in situ carcinoma of uterine cervix.
• Participants who must or wish to continue the intake of restricted medications or any drug considered likely to interfere with the safe conduct of the trial.
• Participants with uncontrolled IIM manifestations that, in the opinion of the investigator, would be likely to require treatment with restricted medications (e.g. cyclophosphamide) during the trial.
• Participants diagnosed with advanced interstitial lung disease (ILD), with:
• Forced vital capacity (FVC) \<70% of predicted at screening OR
• A history of any of the following up to 12 months prior to screening:
• Diffusing capacity of the lung for carbon monoxide (DLCO) \<70% of predicted
• Hypoxemia requiring supplemental oxygen therapy, not attributable to sleep apnoea OR
• Recent ILD exacerbation or respiratory hospitalisation related to ILD within the last 3 months.
• Participants who experience improvement in MMT-8 (\>10%) or PhGA (≥2 cm) from screening (Visit 1) to randomisation (Visit 2).
• Severe muscle damage defined as a global muscle damage score of \>5 on a 10-centimetre visual analogue scale (VAS) scale on the myositis damage Index (MDI).

Sponsors & Collaborators

• Boehringer Ingelheim: lead

Study Sites (138)

Arizona Neuromuscular Research Center

Phoenix, Arizona, 85028, United States

Location

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

University of California Irvine

Irvine, California, 92697, United States

Location

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

University of California San Francisco

San Francisco, California, 94115, United States

Location

Heuer M.D. Research, Inc

Orlando, Florida, 32819, United States

Location

Kansas University Medical Center

Fairway, Kansas, 66205, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21224, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109-5314, United States

Location

Mayo Clinic, Rochester

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Paramount Medical Research and Consulting

Middleburg Heights, Ohio, 44130, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

West Tennessee Research Institute

Jackson, Tennessee, 38305, United States

Location

Helios CR, Inc - Dallas

Dallas, Texas, 75231, United States

Location

Nerve and Muscle Center of Texas

Houston, Texas, 77030, United States

Location

Bioclinic Research

C.a.b.a, C1426BOD, Argentina

Location

Centro Argentino de Investigacion (CADI)-Diagnostico Medico

CABA, C1113, Argentina

Location

Instituto de Investigacion Clinica TyT

CABA, C1405BFN, Argentina

Location

Instituto Medico Especializado

Capital Federal, C1405BCH, Argentina

Location

Framingham Centro Medico

La Plata, B1902COS, Argentina

Location

Instituto Medico CER

Quilmes, B1878DVB, Argentina

Location

Sanatorio de la Mujer

Rosario, S2000ORE, Argentina

Location

Centro de Investigaciones Medicas Tucuman

San Miguel de Tucumán, 4000, Argentina

Location

Alto Dorrego Consultorios

Villa Nueva, M5526DRW, Argentina

Location

Wollongong Hospital

Wollongong, New South Wales, 2500, Australia

Location

Austin Health

Ivanhoe, Victoria, 3079, Australia

Location

Universitair Ziekenhuis Brussel

Jette, 1090, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Centre Hospitalier Universitaire de Liège

Liège, 4000, Belgium

Location

Centro Mineiro de Pesquisa (CMIP)

Juiz de Fora, 36010-570, Brazil

Location

Irmandade da Santa Casa de Misericórdia de Porto Alegre

Porto Alegre - RS, CEP 90020-090, Brazil

Location

Hospital Mater Dei BA

Salvador, 40221-500, Brazil

Location

Hospital das Clínicas da Faculdade de Medicina da USP

São Paulo, 01246-903, Brazil

Location

Azidus Centro de Pesquisa Clinica Avancada

Valinhos, 13271-130, Brazil

Location

Medical Center MedTech Services Ltd.

Haskovo, 6304, Bulgaria

Location

Alexandrovska University Hospital

Sofia, 1431, Bulgaria

Location

University Multiprofile Hospital for Active Treatment St. Ivan Rilski EAD

Sofia, 1612, Bulgaria

Location

China-Japan Friendship Hospital

Beijing, 100029, China

Location

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Second Affiliated Hospital Chongqing Medical University

Chongqing, 400010, China

Location

Dongguan People's Hospital

Dongguan, 523059, China

Location

Guangdong Provincial People's Hospital

Guangzhou, 510080, China

Location

The third affiliated hospital of Sun Yat-Sen University

Guangzhou, 510630, China

Location

Anhui Provincial Hospital

Hefei, 230001, China

Location

The First Affiliated Hospital of Henan University of Science and Technology

Luoyang, 471003, China

Location

The Second Affiliated Hospital to Nanchang University

Nanchang, 330038, China

Location

Nanjing Drum Tower Hospital

Nanjing, 210008, China

Location

Ruijin Hospital Shanghai Jiao Tong University School of Medicine

Shanghai, 200025, China

Location

Huashan Hospital, Fudan University

Shanghai, 200040, China

Location

Renji Hospital Shanghai Jiaotong University School of Medicine

Shanghai, 201112, China

Location

The First Affiliated Hospital of Wenzhou Medical University

Wenzhou, 325000, China

Location

Tongji Hospital Affiliated Tongji Medical College Huazhong University of S & T

Wuhan, 430030, China

Location

First Affiliated Hospital of Xi'an JiaoTong University

Xi'an, 710061, China

Location

HOP d'Angers

Angers, 49933, France

Location

HOP Pellegrin

Bordeaux, 33076, France

Location

HOP CHU Caen

Caen, 14000, France

Location

Hôpital Edouard Herriot

Lyon, 69437, France

Location

HOP Saint-Louis

Paris, 75010, France

Location

Hôpital de la Pitié Salpêtrière

Paris, 75013, France

Location

HOP Civil

Strasbourg, 67091, France

Location

Hôpital Rangueil - CHU de Toulouse

Toulouse, 31059, France

Location

Auenlandklinik Bad Bramstedt GmbH

Bad Bramstedt, 24576, Germany

Location

Immanuel-Krankenhaus GmbH

Berlin, 13125, Germany

Location

Krankenhaus Porz am Rhein gGmbH

Cologne, 51149, Germany

Location

Universitätsklinikum Erlangen

Erlangen, 91054, Germany

Location

Universitätsklinikum Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Martin-Luther-Universität Halle-Wittenberg

Halle, 06120, Germany

Location

medius Kliniken gGmbH

Kirchheim unter Teck, 73230, Germany

Location

Universitätsklinikum Tübingen

Tübingen, 72076, Germany

Location

Universitätsklinikum Ulm

Ulm, 89081, Germany

Location

General Hospital of Thessaloniki "Ippokrateio"

Thessaloniki, 546 42, Greece

Location

University of Debrecen

Debrecen, 4032, Hungary

Location

University of Pecs

Pécs, 7634, Hungary

Location

Azienda Universitaria Ospedaliera Consorziale Policlinico Bari

Bari, 70124, Italy

Location

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

Catania, 95121, Italy

Location

Azienda Ospedaliera di Padova

Padova, 35100, Italy

Location

Fondazione IRCCS Policlinico S. Matteo

Pavia, 27100, Italy

Location

Azienda Ospedaliera Universitaria Pisana

Pisa, 56126, Italy

Location

Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino

Torino, 10126, Italy

Location

Japan Community Healthcare Organization Chukyo Hospital

Aichi, Nagoya, 457-8510, Japan

Location

Hospital of the University of Occupational and Environmental Health

Fukuoka, Kitakyushu, 807-8556, Japan

Location

Fukushima Medical University Hospital

Fukushima, Fukushima, 960-1295, Japan

Location

Hokkaido University Hospital

Hokkaido, Sapporo, 060-8648, Japan

Location

St. Marianna University Hospital

Kanagawa, Kawasaki, 216-8511, Japan

Location

Yokohama City University Hospital

Kanagawa, Yokohama, 236-0004, Japan

Location

Kyoto University Hospital

Kyoto, Kyoto, 606-8507, Japan

Location

Tohoku Medical and Pharmaceutical University Hospital

Miyagi, Sendai, 983-8512, Japan

Location

Nara Medical University Hospital

Nara, Kashihara, 634-8522, Japan

Location

Okayama University Hospital

Okayama, Okayama, 700-8558, Japan

Location

The University of Osaka Hospital

Osaka, Suita, 565-0871, Japan

Location

Juntendo University Hospital

Tokyo, Bunkyo-ku, 113-8431, Japan

Location

Institute of Science Tokyo Hospital

Tokyo, Bunkyo-ku, 113-8519, Japan

Location

Nippon Medical School Hospital

Tokyo, Bunkyo-ku, 113-8603, Japan

Location

Toho University Omori Medical Center

Tokyo, Ota-ku, 143-8541, Japan

Location

Tokyo Medical University Hospital

Tokyo, Shinjuku-ku, 160-0023, Japan

Location

Keio University Hospital

Tokyo, Shinjuku-ku, 160-8582, Japan

Location

Tokyo Women's Medical University Hospital

Tokyo, Shinjuku-ku, 162-8666, Japan

Location

Mediadvance Clinical S.A.P.I de C.V.

Chihuahua City, 31203, Mexico

Location

Centro Integral en Reumatologia, SA. de CV.

Guadalajara, 44160, Mexico

Location

Panamerican Clinical Research Mexico

Guadalajara, 44670, Mexico

Location

CIMOVA (Consultorio anexo)

León, 37000, Mexico

Location

CITER Centro de Investigación y Tratamiento de las Enfermedades Reumaticas SA de CV

Mexico City, 06700, Mexico

Location

Inst Nac de Ciencias Medicas y Nutricion Salvador Zubiran

Mexico City, 14080, Mexico

Location

Medical Care & Research SA de CV

Mérida, 97070, Mexico

Location

Amsterdam UMC Locatie AMC

Amsterdam, 1105 AZ, Netherlands

Location

Nova Reuma Domyslawska I Rusilowicz - Spolka Partnerska Lekarza Reumatologa I Fizjoterapeuty

Bialystok, 15-707, Poland

Location

Szpital Uniwersytecki Nr 2 Im Dr Jana Biziela W Bydgoszczy

Bydgoszcz, 85-168, Poland

Location

Gornoslaskie Centrum Medyczne im prof. Leszka Gieca Slaskiego Uniwersytetu Medycznego W Katowicach

Katowice, 40635, Poland

Location

ULS da Região de Aveiro

Aveiro, 3814-501, Portugal

Location

CHUC - Centro Hospitalar e Universitario de Coimbra, EPE

Coimbra, 3004-561, Portugal

Location

ULS de Santa Maria, E.P.E

Lisbon, 1649-035, Portugal

Location

Spitalul Clinic Dr. I. Cantacuzino

Bucharest, 020475, Romania

Location

Centrul Clinic De Boli Reumatismale Dr. Ion Stoia

Bucharest, 020983, Romania

Location

Spitalul Clinic Judetean de urgenta Cluj

Cluj-Napoca, 400006, Romania

Location

Institute of Rheumatology

Belgrade, 11000, Serbia

Location

Military Medical Academy

Belgrade, 11040, Serbia

Location

University Clinical Center of Kragujevac

Kragujevac, 34000, Serbia

Location

Chonnam National University Hospital

Gwangju, 61453, South Korea

Location

Kyung Hee University Hospital

Seoul, 02447, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Hanyang University Medical Center

Seoul, 04763, South Korea

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Clínic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Santa Creu i Sant Pau

Barcelona, 08041, Spain

Location

Hospital Universitario De La Princesa

Madrid, 28006, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario Virgen de la Victoria

Málaga, 29010, Spain

Location

Hospital Dr. Peset

Valencia, 46017, Spain

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, 80756, Taiwan

Location

Chung Shan Medical University Hospital

Taichung, 402, Taiwan

Location

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

Location

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

Location

Siriraj Hospital

Bangkok, 10700, Thailand

Location

Maharaj Nakom Chiangmai Hospital

Chiang Mai, 50200, Thailand

Location

Songklanagarind Hospital

Hat Yai, 90110, Thailand

Location

Ramathibodi Hospital

Ratchathewi, 10400, Thailand

Location

Related Links

• Related Info

MeSH Terms

Conditions

Myositis

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

Central Study Contacts

Boehringer Ingelheim

CONTACT

1-800-243-0127; clintriage.rdg@boehringer-ingelheim.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2026
• First Posted: August 27, 2026
• Study Start (Estimated): November 9, 2026
• Primary Completion (Estimated): March 21, 2030
• Study Completion (Estimated): March 26, 2030
• Last Updated: August 27, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Time Frame: One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program.
• Access Criteria: For study documents -upon signing of a 'Document Sharing Agreement'. For study data -1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement.

Locations

US (17); BR (5); CN (16); ME (7); SE (3); PL (3); PT (3); ES (7); AR (9); BE (3); KR (4); GR (1); NL (1); FR (8); AU (2); DE (9); HU (2); IT (6); RO (3); BU (3); JP (18); TW (4); TH (4)