Phase ll Study of Pirfenidone in Patients With RAILD (TRAIL1)
- Conditions
- Rheumatoid Arthritis Interstitial Lung Disease
- Interventions
- Drug: Placebo
- Registration Number
- NCT02808871
- Lead Sponsor
- Brigham and Women's Hospital
- Brief Summary
The purpose of this study is to to assess the safety and tolerability of pirfenidone 2403 mg/day for the treatment of RA-associated interstitial lung disease.
- Detailed Description
This is a phase 2, randomized, double blind, placebo controlled trial of pirfenidone for the treatment of RA associated interstitial lung disease. Approximately 270 subjects will be randomized to receive Pirfenidone 2403 mg per day or placebo in a 1:1 ratio. The primary outcome of this study is to assess the efficacy of pirfenidone 2403 mg/day versus placebo in patients with RA associated interstitial lung disease, as defined by progression free survival over the 52 weeks of treatment. Patients will receive blinded study treatment from the time of randomization until the Week 52 Visit.
Eligible patients aged 18 to 85 years must meet 2010 ACR/EULAR criteria for RA (Aletaha, Neogi et al. 2010) as well as RA-associated ILD, as determined by imaging and, when available, lung biopsy. Patients will be required to have a % predicted FVC ≥40 and % predicted DLCO or TLCO ≥30 at screening.
The dose of study treatment will be titrated over 14 days. Patients will receive a telephone assessment at Weeks 1 and 2, and visit the clinic at Weeks 4, 8, 13, 19, 26, 39, and 52. Subjects will have a follow up phone call 28 days after completion of the study drug. Patients should complete a compliance diary between visits. If patients discontinue study treatment for any reason before the end of the study, they should continue with all scheduled study procedures through Week 52. If subjects are unable to complete the study visits as scheduled, all efforts should be made to complete an early termination visit.
The primary outcome variable of this study will be progression free survival, defined as progression free from decline in FVC of 10% or greater during the 52 week study period.
More information can be found at www.ralung.org.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 123
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Placebo for 52 weeks Pirfenidone Pirfenidone Pirfenidone 2403 mg/d for 52 weeks
- Primary Outcome Measures
Name Time Method Number of Participants Who Developed Any Element of the Composite Endpoint 52 weeks Number of participants who developed any element of the composite endpoint of decline in percent predicted FVC of 10% or greater or death.
- Secondary Outcome Measures
Name Time Method All Cause Hospitalization 52 weeks Number of participants requiring hospitalization for any cause
Acute Exacerbations Requiring Hospitalization 52 weeks Number of participants experiencing acute exacerbation requiring hospitalization
Treatment-emergent Adverse Events (AEs) 52 weeks Number of participants with treatment-emergent adverse events (AEs)
Hospitalization for Respiratory Cause 52 weeks Number of participants requiring hospitalization for respiratory cause
Number of Participants With FVC Decline From Baseline of 10% or Greater 52 weeks Number of participants with decline from baseline in percent predicted FVC of 10% or greater during the study period.
All-cause Mortality 52 weeks Number of participants experiencing mortality due to all causes
Change in Absolute Value FVC Over the 52 Week Study Period 52 weeks Change from baseline to end of study in absolute value of FVC over the 52 week study period
Change in PRO of Dyspnea 52 weeks Change from Baseline to end of study in dyspnea, as measured by the Dyspnea 12 questionnaire - Total scores range from 0 to 36, with higher scores corresponding to greater severity.
Treatment-emergent/Treatment-related AEs 52 weeks Number of participants with treatment-emergent/treatment-related AEs
Number of Participants With Progressive Disease 52 weeks Number of participants with progressive disease as defined by OMERACT: FVC% relative decline of \>=10% or FVC% change in \>=5\< 10% and \>=15% diffusing capacity (DLCO)
Time to Composite of Decline in FVC or Death 52 weeks Time to decline of 10% or greater in percent predicted FVC or death while on study
Change in % Predicted FVC From Baseline to End of Study Over the 52 Week Study Period 52 weeks Change from baseline to end of study of percent predicted FVC over the 52 week study period
Treatment-emergent Serious Adverse Events (SAEs) 52 weeks Number of participants with treatment-emergent serious adverse events (SAEs) in the as treated population
Treatment-emergent/Treatment-related SAEs 52 weeks Number of participants with treatment-emergent/treatment-related SAEs
AEs Leading to Early Discontinuation of Study Treatment 52 weeks Number of participants with AEs leading to early discontinuation of study treatment
Treatment-emergent Death or Transplant 52 weeks Number of participants who experienced treatment-emergent death or transplant
Treatment-emergent RA-ILD-related Mortality 52 weeks Number of participants who experienced treatment-emergent RA-ILD-related mortality
Trial Locations
- Locations (33)
Weill Cornell Medicine
🇺🇸New York, New York, United States
University of Calgary Cummings School of Medicine
🇨🇦Calgary, Alberta, Canada
Royal Brompton and Harefield NHS Foundation Trust
🇬🇧London, United Kingdom
Newcastle upon Tyne Hospitals NHS Foundation Trust
🇬🇧Newcastle Upon Tyne, United Kingdom
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Manchester University NHS Foundation Trust (South) Wythenshawe Hospita
🇬🇧Manchester, United Kingdom
The Prince Charles Hospital
🇦🇺Camperdown, Australia
Norfolk and Norwich University Hospitals NHS Foundation Trust
🇬🇧Norwich, United Kingdom
Tulane Medical Center
🇺🇸New Orleans, Louisiana, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Alabama Site at Birmingham
🇺🇸Birmingham, Alabama, United States
North Bristol NHS Trust Headquarters, Southmead Hospital
🇬🇧Bristol, United Kingdom
University of California San Francisco
🇺🇸San Francisco, California, United States
University of Utah Health Care
🇺🇸Salt Lake City, Utah, United States
St. Joseph's Healthcare
🇨🇦Hamilton, Ontario, Canada
Royal Prince Alfred Hospital
🇦🇺Camperdown, Sydney, Australia
John Hopkins Medicine
🇺🇸Baltimore, Maryland, United States
National Jewish Health
🇺🇸Denver, Colorado, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Royal Brompton
🇦🇺Brisbane, Queensland, Australia
Melbourne Alfred Hospital
🇦🇺Melbourne, Victoria, Australia
Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
St. Paul's Hospital - Providence Health Care
🇨🇦Vancouver, British Columbia, Canada
Royal Devon and Exeter NHS Foundation
🇬🇧Exeter, United Kingdom
Aintree University Hospitals NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southhampton, United Kingdom
Papworth Hospital NHS Foundation Trust
🇬🇧Cambridge, United Kingdom
University Hospitals of Leicester NHS Foundation Trust
🇬🇧Leicester, United Kingdom
Leeds Teaching Hospitals NHS Trust
🇬🇧Leeds, United Kingdom
Oxford University Hospitals NHS Foundation Trust
🇬🇧Oxford, United Kingdom
University of Miami
🇺🇸Miami, Florida, United States
University of Washington
🇺🇸Seattle, Washington, United States