Clinical trial NCT00080223: Safety Study of Oral Pirfenidone in Patients With Pulmonary Fibrosis/Idiopathic Pulmonary Fibrosis
Official title: An Open-Label, Phase 2 Study of the Safety of Oral Pirfenidone in Patients With Pulmonary Fibrosis/Idiopathic Pulmonary Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Idiopathic Pulmonary Fibrosis
Pulmonary Fibrosis
Keywords: pulmonary fibrosis
respiratory diseases
Lead sponsor: Genentech, Inc.
Sponsor class: INDUSTRY
First posted: 2004-03-26
Enrollment: 83
Sex: ALL
Minimum age (years): 40
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: To assess the safety of treatment with pirfenidone (up to 3600 mg/d) in patients with pulmonary fibrosis/idiopathic pulmonary fibrosis (PF/IPF).

Detailed description: This study has been designed as a rollover study to collectively include safety data from various previous studies.

In addition, InterMune has also initiated an Early Access Program to make pirfenidone available to a limited number of patients with idiopathic pulmonary fibrosis in the United States. This program is also being conducted under this protocol. Registration of patients with documented IPF has been closed as of October 2005.

Eligibility criteria: General Inclusion Criteria:

* Able to understand and sign an informed consent form
* Understand the importance of adherence to study treatment and the study protocol, including concomitant medication restrictions, throughout the study period
* Patients must be willing to travel to an approved regional center for all study-related visits

Roll-Over Criteria:

* Entry into study through rollover has been completed

Criteria for Early Access Program patients:

* Clinical symptoms consistent with IPF ≥3 months duration
* Age 40 - 85, inclusive
* At the time of registration with National Organization for Rare Disorders (NORD), patients with IPF must have a percent predicted forced vital capacity (FVC) of ≥50%, and percent predicted carbon monoxide diffusing capacity (DLCO) of ≥35%
* At the time of enrollment in PIPF-002, (screening/baseline visit) percent predicted FVC must be ≥45%, and percent predicted DLCO must be ≥30%
* High-resolution computed tomographic scan (HRCT) showing definite IPF. For patients with surgical lung biopsy showing definite or probable usual interstitial pneumonia (UIP), the HRCT criterion of probable IPF is sufficient
* For patients aged \<50 years: open or video-assisted thoracoscopic (VATS) lung biopsy showing definite or probable UIP. In addition, no features supporting an alternative diagnosis on transbronchial biopsy or bronchoalveolar lavage if performed
* For patients aged ≥50 years: at least one of the following diagnostic findings as well as the absence of any features on specimens resulting from any of these procedures that support an alternative diagnosis: 1) Open or VATS lung biopsy showing definite or probable UIP; 2) Transbronchial biopsy showing no features to support an alternative diagnosis; 3) Bronchoalveolar lavage (BAL) showing no features to support an alternative diagnosis

Primary outcomes:
measure: Percentage of Participants With a Treatment-Emergent Adverse Event (AE), Serious AE (SAE), Severe AE, Life-threatening AE, Death or Discontinuation Because of an AE
timeFrame: Baseline to 28 days after the last dose of study treatment (maximum duration of treatment in study was 604 weeks)
Secondary outcomes:
measure: Percent Predicted Forced Vital Capacity (FVC)
timeFrame: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, 336, 360, 384, 408, 432, 456, 480
measure: Hemoglobin (Hgb)-Corrected Percent-Predicted Carbon Monoxide Diffusing Capacity (DLco)
timeFrame: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, 336, 360, 384, 408, 432, 456, 480
measure: Resting Oxygen Saturation by Pulse Oximetry (SpO2)
timeFrame: Baseline, Weeks 24, 48, 72, 96, 120, 144, 168, 192, 216, 240, 264, 288, 312, 336, 360, 384, 408, 432, 456, 480
measure: Overall Survival
timeFrame: First dosing of study treatment until death (up to 604 weeks)
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
city: Pomona
country: United States
state: California
city: San Jose
country: United States
state: California
city: New Haven
country: United States
state: Connecticut
city: Sarasota
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
city: Kailua
country: United States
state: Hawaii
city: Lahaina
country: United States
state: Hawaii
city: Nampa
country: United States
state: Idaho
city: Elk Grove Village
country: United States
state: Illinois
city: Boston
country: United States
state: Massachusetts
city: West Roxbury
country: United States
state: Massachusetts
city: St. Louis
country: United States
state: Missouri
city: Huntington Station
country: United States
state: New York
city: New York
country: United States
state: New York
city: New York
country: United States
state: New York
city: Rochester
country: United States
state: New York
city: Worthington
country: United States
state: Ohio
city: Portland
country: United States
state: Oregon
city: Portland
country: United States
state: Oregon
city: Lancaster
country: United States
state: Pennsylvania
city: Dallas
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Provo
country: United States
state: Utah
city: Annandale
country: United States
state: Virginia
city: Bremerton
country: United States
state: Washington
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References:
citation: Gotfried MH, Girod CE, Antin-Ozerkis D, Burgess T, Strombom I, Stauffer JL, Kirchgaessler KU, Padilla ML. An Open-Label, Phase II Study of the Safety of Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis (PIPF-002). Pulm Ther. 2018 Jun;4(1):59-71. doi: 10.1007/s41030-018-0053-y. Epub 2018 Apr 5.