Pirfenidone for Progressive Fibrotic Sarcoidosis (PirFS)

Study comparing pirfenidone versus placebo for patients with advanced fibrotic sarcoidosis

Study Overview

• Status: Unknown
• Conditions: Sarcoidosis, Pulmonary
• Intervention / Treatment: Drug: Pirfenidone; Drug: Placebos

Detailed Description

Patients who meet the inclusion and exclusion criteria will be randomized to be treated with either placebo or pirfenidone at the current approved dosage for idiopathic pulmonary fibrosis at a 2:1 pirfenidone to placebo ratio. Patients treated with pirfenidone will be titrated using the following schedule:

One 267 mg capsules three times a day for two weeks. Two 267 mg capsules three times a day for two weeks Three 267 mg capsules three times a day thereafter Patients will be instructed to take all doses of medication with food. Dosage will be titrated on an individual basis depending on patient tolerance of medication.

Those randomized to placebo will receive the same schedule using placebo tablets.

Block randomization will be done at each site. Liver function tests will be evaluated as listed above. If the patient has an abnormal liver function test, then the dose will be adjusted per the company's protocol for commercial drug.

• Study Type: Interventional
• Enrollment (Anticipated): 60
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Robert P Baughman, MD; Phone Number: 513-584-5225; Email: baughmrp@ucmail.uc.edu
• Study Contact Backup: Name: Rebecca Reeves; Phone Number: 513-584-5226; Email: Rebecca.Klein@UCHealth.com

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Recruiting
      • University of Cincinnati

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 90 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of sarcoidosis
• Pulmonary function testing with a Composite Physiologic Index (CPI) score of greater than 40
• Patient must have evidence of >20% fibrosis on high resolution cat scan
• Patients must be on a stable prednisone therapy for sarcoidosis for at least two months and no change in other immunosuppressives in the two months prior to entry into study
• Age greater than 18 and less than 90.
• Able to provide written informed consent for participation in the study

Exclusion Criteria:

• Patients receiving therapy for precapillary pulmonary hypertension.
• Patients with liver disease Childs class 3 or 4
• Patients with a left ventricular ejection fraction of less than 40%
• Patients receiving more than 20 mg prednisone daily or its equivalent
• Patients with massive hemoptysis within prior three months. Patients with mycetomas are eligible as long as no massive hemoptysis in prior three months.
• Patients with clinically important co-existing disease which in the opinion of the investigator is likely to affect patient's chance for survival during the course of the study
• Patient who is pregnant, lactating, intending to become pregnant during the study, or child bearing capacity who is not willing to use appropriate birth control methods approved by investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Pirfenidone; Pirfenidone titrated to three 267 mg tablets three times a day	Drug: Pirfenidone; Increasing doses; Other Names: esbriet
Placebo Comparator: Placebos; Placebo titrated to three tablets three times a day	Drug: Placebos; Increasing doses; Other Names: placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time until clinical worsening (TCW)	TCW	two years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in forced vital capacity (FVC)	Change in forced vital capacity	two years
Change in CPI	Change in composite physiologic index	two years

Collaborators and Investigators

• Sponsor: University of Cincinnati
• Collaborators: Royal Brompton & Harefield NHS Foundation Trust
• Investigators: Principal Investigator: Robert P Baughman, MD, University of Cincinnati

Study record dates

Study Major Dates

• Study Start (Actual): September 27, 2017
• Primary Completion (Anticipated): December 31, 2019
• Study Completion (Anticipated): March 30, 2020

Study Registration Dates

• First Submitted: August 22, 2017
• First Submitted That Met QC Criteria: August 22, 2017
• First Posted (Actual): August 24, 2017

Study Record Updates

• Last Update Posted (Actual): September 28, 2017
• Last Update Submitted That Met QC Criteria: September 27, 2017
• Last Verified: September 1, 2017

More Information

Terms related to this study

• Keywords: pulmonary fibrosis
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Lung Diseases; Lung Diseases, Interstitial; Sarcoidosis, Pulmonary; Sarcoidosis; Physiological Effects of Drugs; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Antineoplastic Agents; Pirfenidone
• Other Study ID Numbers: 2016-5706

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided
• IPD Plan Description: no plans yet

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes