Clinical trial NCT03260556: Pirfenidone for Progressive Fibrotic Sarcoidosis
Official title: Pirfenidone for Progressive Fibrotic Sarcoidosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Sarcoidosis, Pulmonary
Keywords: pulmonary fibrosis
Lead sponsor: University of Cincinnati
Sponsor class: OTHER
Collaborators: name: Royal Brompton & Harefield NHS Foundation Trust
First posted: 2017-08-24
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Double blind placebo controlled trial
Primary purpose: TREATMENT

Brief summary: Study comparing pirfenidone versus placebo for patients with advanced fibrotic sarcoidosis

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.

Eligibility criteria: 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

Primary outcomes:
measure: Time until clinical worsening (TCW)
timeFrame: two years
Secondary outcomes:
measure: Change in forced vital capacity (FVC)
timeFrame: two years
measure: Change in CPI
timeFrame: two years
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
Study officials:
affiliation: University of Cincinnati
name: Robert P Baughman, MD
role: PRINCIPAL_INVESTIGATOR
References: