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

Phase 3Not Yet RecruitingNCT07789015

Boehringer Ingelheim270 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

270

Conditions

Idiopathic Inflammatory Myopathies

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion criteria: 1. 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. 2. 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. 3. Participants with overlap myositis (OM) must have IIM as the predominant disease. 4. 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. 5. Disease activity defined by moderate to severe myopathy. 6. The participant must be on background therapy for IIM. 7. Woman (or women) of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control for a period of 28 days before treatment initiation, throughout the trial, and for a period of at least 5 days after the last dose of investigational medicinal product (IMP). WOCBP taking oral contraceptives also have to use one barrier method. 8. Signed and dated written informed consent in accordance with ICH-GCP and local legislation prior to admission to the trial. 9. Further inclusion criteria apply. Exclusion criteria: 1. 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. 2. 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. 3. 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. 4. Participants diagnosed with advanced interstitial lung disease (ILD). 5. Severe muscle damage. 6. Further exclusion criteria apply.

Locations (147)

Arizona Neuromuscular Research Center

Phoenix, Arizona, United States

Mayo Clinic

Scottsdale, Arizona, United States

University of California Irvine

Irvine, California, United States

University of California Los Angeles

Los Angeles, California, United States

University of California San Francisco

San Francisco, California, United States

Outcomes

Primary Outcomes

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.

Time frame: At Week 52.

Secondary Outcomes

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

Number of participants with 40 TIS points or higher.

Time frame: At Week 52.

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

Number of participants achieving a TIS-40 response at Week 52 and ≤5 mg/day of prednisone (or equivalent) at Week 52

Time frame: 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)

The Cutaneous Dermatomyositis Disease Area and Severity Index activity score (CDASI-A) is a questionnaire assessing skin disease activity and damage in patients with dermatomyositis (DM). It ranges from 0 to 100, with higher values indicating more severe active skin involvement.