A Study of Pirfenidone in Patients With Unclassifiable Progressive Fibrosing Interstitial Lung Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03099187
• Date of registration: 31/03/2017
• Prospective Registration: Yes
• Primary sponsor: Hoffmann-La Roche
• Public title: A Study of Pirfenidone in Patients With Unclassifiable Progressive Fibrosing Interstitial Lung Disease
• Scientific title: Multicenter, International, Double-blind, Two-Arm, Randomized, Placebo-controlled Phase II Trial of Pirfenidone in Patients With Unclassifiable Progressive Fibrosing ILD
• Date of first enrolment: May 15, 2017
• Target sample size: 253
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03099187
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Canada, Czechia, Denmark, Germany, Greece, Ireland, Israel, Italy, Poland, Portugal, Spain, United Kingdom

Contacts

• Name: Clinical Trials
• Affiliation: Hoffmann-La Roche

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age >= 18-85 years

- Confirmed fibrosing ILD which, following multidisciplinary team review, cannot be
classified with either high or moderate confidence as a specific idiopathic
interstitial pneumonia or other defined ILD

- Progressive disease as considered by the investigator as participants deterioration
within the last 6 months, which is defined as a rate of decline in forced vital
capacity (FVC) >5% or a significant symptomatic worsening not due to cardiac,
pulmonary vascular or other causes

- Extent of fibrosis >10% on high-resolution computed tomography

- Forced vital capacity >= 45% of predicted value

- Diffusing capacity of the lung for carbon monoxide (DLco) >= 30% of predicted value

- Forced expiratory volume in 1 second/FVC ratio >= 0.7

- Able to do 6-minute walk distance (6MWD) >= 150 meters

- For women of childbearing potential: agreement to remain abstinent or use a
non-hormonal or hormonal contraceptive method with a failure rate of < 1% per year
during the treatment period and for at least 90 days after the last dose of
pirfenidone

- For men, agreement to remain abstinent or use contraceptive measures, and agreement to
refrain from donating sperm

Exclusion Criteria:

- Diagnosis with moderate or high confidence of nonspecific interstitial pneumonia and
any ILD with an identifiable cause such as connective tissue disease-ILD, chronic
hypersensitivity pneumonitis, or others

- Diagnosis of idiopathic pulmonary fibrosis independent of the confidence level

- History of unstable angina or myocardial infarction during the previous 6 months

- Treatment with high dose systemic corticosteroids, or any immunosuppressant other than
mycophenolate mofetil/acid (MMF), at any time within the 4 weeks of the screening
period. Participants being treated with MMF should be on a stable dose that is
expected to remain stable throughout the trial and was started at least 3 months prior
to screening

- Participants previously treated with pirfenidone or nintedanib

- Participants treated with N-acetyl-cysteine for fibrotic lung disease, at any time
within the 4 weeks of the screening period

- Drug treatment for any type of pulmonary hypertension

- Participation in a trial of an investigational medicinal product within the last 4
weeks

- Significant other organ co-morbidity including hepatic or renal impairment

- Predicted life expectancy < 12 months or on an active transplant waiting list

- Use of any tobacco product in the 12 weeks prior to the start of screening, or any
unwillingness to abstain from their use through to the Follow-up Visit

- Illicit drug or alcohol abuse within 12 months prior to screening

- Planned major surgery during the trial

- Hypersensitivity to the active substance or to any of the excipients of pirfenidone

- History of angioedema

- Concomitant use of fluvoxamine

- Clinical evidence of any active infection

- Any history of hepatic impairment, elevation of transaminase enzymes, or liver
function test results as: Total bilirubin above the upper limit of normal (ULN),
Aspartate aminotransferase or alanine aminotransferase >1.5 × ULN, and Alkaline
phosphatase >2.0 × ULN

- Creatinine clearance < 30 milliliter (mL) per minute, calculated using the
Cockcroft-Gault formula

- Any serious medical condition, clinically significant abnormality on an
Electrocardiogram (ECG) at screening, or laboratory test results

- An ECG with a heart rate corrected QT interval using Fridericia's formula as >= 500
milliseconds at screening, or a family or personal history of long QT syndrome



Age minimum: 18 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Lung Diseases, Interstitial

Intervention(s)

Drug: Pirfenidone

Drug: Placebo

Primary Outcome(s)

Rate of Decline in Forced Vital Capacity (FVC) Over the 24-week Double-blind Treatment Period [Time Frame: Up to Week 24]

Secondary Outcome(s)

Categorical Change in FVC of >10% [Time Frame: Baseline (Day 1) to Week 24]

Number of Participants With Dose Reductions and Treatment Interruptions [Time Frame: From administration of the first dose of study drug to Week 24]

Change in Percent Predicted FVC [Time Frame: Baseline (Day 1) to Week 24]

Categorical Change in FVC of >5% [Time Frame: Baseline (Day 1) to Week 24]

Percentage of Participants With Investigator-reported Acute Exacerbations [Time Frame: Baseline (Day 1) to Week 24]

Progression-free Survival (PFS) [Time Frame: Baseline (Day 1) to Week 24]

Change in FVC [Time Frame: Baseline (Day 1) to Week 24]

Change in Score in Leicester Cough Questionnaire Score [Time Frame: Baseline (Day 1) to Week 24]

Change in Cough Visual Analog Scale (VAS) Score [Time Frame: Baseline (Day 1) to Week 24]

Change in University of California, San Diego-Shortness of Breath Questionnaire Score [Time Frame: Baseline (Day 1) to Week 24]

Change in Total and Sub-scores of the Saint George's Respiratory Questionnaire (SGRQ) [Time Frame: Baseline (Day 1) to Week 24]

Number of Participants Withdrawn From Trial Treatment or Trial Discontinuations [Time Frame: Baseline (Day 1) to Week 24]

Number of Participants With Non-elective Hospitalization, Both Respiratory and All Cause [Time Frame: Baseline (Day 1) to Week 24]

Time to Death From Respiratory Diseases [Time Frame: Baseline (Day 1) to Week 24]

Time to First Investigator-reported Acute Exacerbations [Time Frame: Baseline (Day 1) to Week 24]

Number of Participants With Treatment-emergent Adverse Events (TEAEs) [Time Frame: Baseline (Day 1) to Week 28]

Change in 6-minute Walk Distance (6MWD) [Time Frame: Baseline (Day 1) to Week 24]

Change in Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) [Time Frame: Baseline (Day 1) to Week 24]

Time to Death From Any Cause [Time Frame: Baseline (Day 1) to Week 24]

Secondary ID(s)

2016-002744-17

MA39189

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 03/01/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03099187