Progressive Fibrosing Interstitial Lung Disease Clinical Trial
Official title:
A Double Blind, Randomized, Placebo-controlled Trial Evaluating the Efficacy and Safety of Nintedanib Over 52 Weeks in Patients With Progressive Fibrosing Coal Mine Dust-Induced Interstitial Lung Disease (PF-CMD-ILD)
Assess efficacy (as measured by annual rate of decline in FVC) and safety. The hypothesis is that nintedanib will be safe and effective therapy for patients with progressive fibrosing CMD-ILD over a period of 52 weeks. Test Article - Nintedanib 150 mg administered PO twice daily or matching placebo. A total of 160 patients meeting inclusion/exclusion criteria will be randomized in a 1:1 ratio to either oral nintedanib 150 mg (n=80) or matching placebo (n=80) twice daily. A randomization scheme will be used that balances the group for potential confounders (proportion with PMF or small opacity-only PF-CMD_ILD and proportion of ever- or never-smokers). The dose of the study drug may be reduced to 100 mg twice daily or interrupted temporarily to manage adverse events (AEs).
This is a single center, prospective, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of nintedanib in patients with progressive fibrosing CMD-ILD over 52 weeks. The study will be led by and based at the West Virginia University School of Medicine and the West Virginia Clinical and Translational Science Institute (WVCTSI) and will recruit participants regionally, including from its associated Practice-Based Network of 24 healthcare systems and 94 individual clinics. Recruitment will also be sought in collaboration with Dr. Cohen from federally-funded Black Lung Clinics across the region and from partner CTSAs at the University of Kentucky and University of Virginia. It is planned to randomize a total of 160 patients in a 1:1 ratio to either oral nintedanib 150 mg (n=80) or matching placebo (n=80) twice daily. The dose of the study drug may be reduced to 100 mg twice daily with subsequent increase back to full dose or interrupted temporarily to manage adverse events (AEs). For each patient, the study will consist of two parts: part A and part B. The duration of part A, a randomized prospective controlled double-blinded trial, will be 52 weeks. Following completion of the week 52 visit, patients will continue to have study visits every 16 weeks (Part B) until the last patient completes 52 weeks. In part B, patients will remain on blinded therapy with continued data collection. In part A, patients will attend screening study visits (-4 to -2 weeks) and then at 0, 4, 8, 12, 24, 36, and 52 weeks. In addition, telehealth visits will be conducted at visits 2, 18, 30, 44, and 51 weeks via phone by the study coordinator. Part B visits will take place at 16 week intervals until study completion (last patient completes 52 weeks). To reduce the amount of missing data, patients who discontinue the trial drug (for any reason) prior to completing the 52-week treatment period will be asked to attend all visits and undergo all examinations as originally planned. In addition, for patients who prematurely discontinue trial medication and are unable to complete the scheduled visits, every attempt will be made to collect information on vital status at week 52 and at the time of data cut-off for the primary data analysis and at the conclusion of part B. It is estimated that patient recruitment will take up to 24 mos. ;