Tolerability and Safety of Nintedanib in Myositis Associated Interstitial Lung Disease

There is likely a role for using anti-fibrotic medications in patients with myositis-associated interstitial lung disease (MA-ILD) to slow down disease progression, especially in patients who have fibrotic and progressive disease. These patients however are currently being excluded from clinical trials of anti-fibrotic agents in progressive ILD because of the concomitant use of immunosuppression. The benefit of anti-fibrotic agents is being assessed in other rheumatic diseases and should be assessed in MA-ILD as well. They are a unique group of patients with a heterogeneous disease, and are much more frequently on concomitant immune-modulating therapy. As such, they should be studied on their own in separate clinical trials, and the use of nintedanib should be studied as an addition to standard of care immunosuppression.

The objective of this study is to assess safety and tolerability of nintedanib in patients with MA-ILD.

Study Overview

• Status: Terminated
• Conditions: Interstitial Lung Disease; Myopathy, Inflammatory
• Intervention / Treatment: Drug: Nintedanib 150 milligrams [Ofev]

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montreal, Quebec, Canada, H4A3J1
      • Research Institute McGill University Health Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 1. 18 years and older 2. Diagnosis of autoimmune myopathy (dermatomyositis, polymyositis, overlap myositis or anti-synthetase syndrome) as diagnosed by a rheumatologist.

  3. Interstitial lung disease confirmed by high resolution CT scan (Extent of disease 10% or more on CT done within 12 months of enrolment) with evidence of fibrosis, defined as reticular abnormality with traction bronchiectasis with or without honeycombing.

  4. Evidence of progressive disease within 24 months of screening visit:

  1. Clinically significant decline in Forced Vital Capacity (FVC) % pred based on a relative decline of >=10%
  2. Marginal decline in FVC % pred based on a relative decline of .>=5-<10% combined with worsening of respiratory symptoms
  3. Marginal decline in FVC % pred based on a relative decline of >=5-<10% combined with increasing extent of fibrotic changes on chest imaging
  4. Worsening of respiratory symptoms such as cough or shortness of breath as well as increasing extent of fibrotic changes on chest imaging as per radiologist or pulmonologist who read the scan 5. Current and ongoing treatment with immunosuppressive medications, on a stable medication regimen and dosage for at least 6 weeks (considered standard of care medical therapy) Concomitant medications allowed are:
  1. mycophenolate,
  2. azathioprine,
  3. tacrolimus,
  4. cyclosporine,
  5. rituximab (injection within the last year),
  6. prednisone low dose =<20 mg daily,
  7. Intravenous immunoglobulins

Exclusion Criteria:

1. Contraindication to treatment with nintedanib (based on Canadian labeling)
2. The female patient is pregnant, plans to become pregnant during the course of the study, or is breastfeeding.
3. The male patient plans to father a child during the course of the study
4. Hypersensitivity to nintedanib, peanut or soy
5. Elevated liver enzymes greater than 1.5 times the upper limit of normal
6. Creatinine clearance <30 mL/min
7. Patient with risks factors of aneurysm or artery dissection, such as known history of aneurysm or uncontrolled hypertension

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Nintedanib treatment; Single arm treatment with nintedanib	Drug: Nintedanib 150 milligrams [Ofev]; All patients will be given nintedanib 150 milligrams orally twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tolerability - completed doses	Percentage of subjects who complete 24 weeks on nintedanib. Subjects will be considered to have completed the 24 weeks of the study if they took 90% of the study drug doses.	24 weeks
Safety and adverse events	numbers of patients with adverse events during course of the study	24 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in forced vital capacity	24 weeks
Change in diffusion capacity of the lung for carbon monoxide	24 weeks
Change in 6 minute walking distance	24 weeks

Collaborators and Investigators

• Sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre
• Investigators: Principal Investigator: Deborah Assayag, MD, Research Institute - McGill University Health Center

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2021
• Primary Completion (Actual): December 31, 2024
• Study Completion (Actual): April 30, 2025

Study Registration Dates

• First Submitted: April 13, 2022
• First Submitted That Met QC Criteria: April 13, 2022
• First Posted (Actual): April 19, 2022

Study Record Updates

• Last Update Posted (Estimated): September 2, 2025
• Last Update Submitted That Met QC Criteria: August 25, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Respiratory Tract Diseases; Lung Diseases; Lung Diseases, Interstitial; Myositis; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; nintedanib
• Other Study ID Numbers: 2020-5543

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No