Clinical trial NCT05335278: Tolerability and Safety of Nintedanib in Myositis Associated Interstitial Lung Disease: a Pilot Study
Official title: Tolerability and Safety of Nintedanib in Myositis Associated Interstitial Lung Disease: a Pilot Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Interstitial Lung Disease
Myopathy, Inflammatory
Lead sponsor: McGill University Health Centre/Research Institute of the McGill University Health Centre
Sponsor class: OTHER
First posted: 2022-04-19
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single group, open label study
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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:

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  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

Primary outcomes:
measure: Tolerability - completed doses
timeFrame: 24 weeks
measure: Safety and adverse events
timeFrame: 24 weeks
Secondary outcomes:
measure: Change in forced vital capacity
timeFrame: 24 weeks
measure: Change in diffusion capacity of the lung for carbon monoxide
timeFrame: 24 weeks
measure: Change in 6 minute walking distance
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Montréal
country: Canada
facility: Research Institute McGill University Health Center
state: Quebec
Study officials:
affiliation: Research Institute - McGill University Health Center
name: Deborah Assayag, MD
role: PRINCIPAL_INVESTIGATOR
References: