Clinical trial NCT04461587: Examination of Pirfenidone (Esbriet®) Therapy in Coal Workers' Pneumoconiosis With Pulmonary Fibrosis
Official title: Examination of Pirfenidone (Esbriet®) Therapy in Coal Workers' Pneumoconiosis With Pulmonary Fibrosis Associated With Radiographic and Functional Impairment to Examine the Reduction in Progression of Disease, Reduction of Exacerbation Rates and Possible Reduction of Inflammatory Biomarkers as an Index of Clinical Response.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Coal Workers' Pneumoconiosis (Complicated)
Keywords: Black Lung
Black Lung Disease
Coal Miner's Lung
Coal Worker's Pneumoconiosis
Coalworker's Pneumoconiosis
Pneumoconiosis, Coal Worker's
Lead sponsor: Pulmonary Research of Abingdon, LLC
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2020-07-08
Enrollment: 50
Sex: ALL
Minimum age (years): 40
Maximum age (years): 79
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open label interventional study observing US FDA approved Esbriet (for Interstitial Pulmonary Fibrosis) in Coal Workers' Pneumoconiosis patients with pulmonary fibrosis.
Primary purpose: TREATMENT

Brief summary: Examination of pirfenidone (Esbriet®) therapy in coal workers' pneumoconiosis (black lung) with pulmonary fibrosis (scarring of the lung).

Detailed description: Examination of pirfenidone (Esbriet®) therapy in coal workers' pneumoconiosis with pulmonary fibrosis associated with radiographic and functional impairment to examine the reduction in progression of disease, reduction of exacerbation rates and possible reduction of inflammatory biomarkers as an index of clinical response.

Eligibility criteria: Inclusion Criteria:

* 1. Able and willing to provide informed consent and to comply with study protocol
* 2. Coal miners between the ages 40 to 79 years of age at visit 1with a minimum of 15 years in a coal mining environment
* 3. Chest CT Scan or Chest Xray (within 6 months) confirming diagnosis of CWP, complicated black lung disease/progressive massive fibrosis will be recorded. If CT not available must be obtained at Visit 1.
* 4. Evidence of Loss of lung function defined by decline in FEV1 or FVC or DLCO of 5% within the past 36 months prior to enrollment.
* 5. FEV1 ≤ 75% or FVC ≤ 80% or DLCO ≤ 70% or abnormal 6MWT with oxygen desaturation of 4% or greater than resting at screening or within past 6 months.
* 6. Former smokers and current smokers will be enrolled if documented evidence of CWP/PMF is present and meet PFT guidelines. Current smokers will be given smoking cessation counseling at each visit due to the effect on pirfenidone concentration and efficacy.

Exclusion Criteria:

* 1. History of prior therapy with Esbriet or Ofev (pirfenidone or nintedanib)
* 2. Recent hospitalization (within 30 days prior to screening) for respiratory decompensation.
* 3. Patients using monoclonal antibody therapy or immunosuppressive therapy for other disease process will be excluded, except for the following:

  * Daily prednisone up to 10 mg daily (prescribed for lung disease or rheumatoid arthritis) will be permitted. Chronic use for 3 months prior to enrollment will be documented.
  * Methotrexate for rheumatoid arthritis will be permitted as long as chronic use for 6 months prior to enrollment is documented.
* 4. Elevation of liver function test at screening documenting AST, ALT or total bilirubin \> 3 x ULN or ALP \> 2.5 x ULN.
* 5. Patients who have other pulmonary pathology such as lung cancer, active tuberculosis or atypical mycobacterial infection requiring treatment.
* 6. Patients with recurrent malignancy requiring chemotherapy or radiation therapy. Individuals with a prior diagnosis of localized skin cancer, prostate cancer, localized bladder cancer will not be excluded unless undergoing active treatment. Patients with a prior diagnosis of malignancy treated greater than 5 years ago will be considered for enrollment.
* 7. History of alcohol or drug abuse that would impair or risk the patient's full participation in the drug study in the opinion of the investigator
* 8. Use of any investigational therapy within 4 weeks of enrollment.
* 9. Individuals with clinically significant unstable cardiac disease (ejection fraction ≤35%) or complex arrhythmias per PI discretion. Chronic atrial fibrillation will be permitted if heart rate is controlled. Historical values of ejection fraction will be accepted.
* 10. Individuals with poorly controlled diabetes per PI discretion (Hemoglobin A1c \>9). Historical values will be accepted.
* 11. Pregnancy or lactation.

Primary outcomes:
measure: Forced Vital Capacity (FVC)
timeFrame: 12 months minimum
Secondary outcomes:
measure: Forced Expiratory Volume in 1 second (FEV1)
timeFrame: 12 months minimum
measure: Diffusing capacity of the lung for carbon monoxide (DLCO)
timeFrame: 12 months minimum
measure: 6 minute walk test
timeFrame: 12 months minimum
measure: Chest CT
timeFrame: 12 months
measure: Inflammatory biomarkers
timeFrame: 12 months minimum
measure: St. George's Respiratory Questionnaire (SGRQ)
timeFrame: 12 months minimum
Other outcomes:
measure: Rates of Respiratory Exacerbation
timeFrame: 12 months minimum
Locations:
city: Abingdon
country: United States
facility: Pulmonary Research of Abingdon, LLC
state: Virginia
Study officials:
affiliation: Pulmonary Research of Abingdon, LLC
name: Emory Robinette, MD
role: PRINCIPAL_INVESTIGATOR
References: