Trial Outcomes & Findings for Open-label Study to Assess the Effectiveness of Pirfenidone in Participants With Idiopathic Pulmonary Fibrosis (IPF). (NCT NCT03208933)

Results Overview

FVC is a standard pulmonary function test. FVC is defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Baseline FVC will be the average of the highest FVC measurement recorded at the Screening and Day 1. The FVC at Week 26 will be the average of the highest FVC measurement recorded on two separate days at Week 26.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

60 participants

Primary outcome timeframe

Baseline, Week 26

Results posted on

2020-11-20

Participant Flow

The study enrolled participants in Russia.

Participant milestones

Participant milestones
Measure	Pirfenidone Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Overall Study STARTED	60
Overall Study COMPLETED	47
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Reasons for withdrawal
Measure	Pirfenidone Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Overall Study Death	3
Overall Study Withdrawal by Subject	3
Overall Study Adverse Event	1
Overall Study Lost to Follow-up	2
Overall Study Physician Decision	3
Overall Study Protocol Violation	1

Baseline Characteristics

Open-label Study to Assess the Effectiveness of Pirfenidone in Participants With Idiopathic Pulmonary Fibrosis (IPF).

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pirfenidone n=60 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Age, Continuous	67.4 Years STANDARD_DEVIATION 7.75 • n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	41 Participants n=5 Participants
Race/Ethnicity, Customized Russian	48 Participants n=5 Participants
Race/Ethnicity, Customized Tatar	4 Participants n=5 Participants
Race/Ethnicity, Customized Bashkir	4 Participants n=5 Participants
Race/Ethnicity, Customized Armenian	1 Participants n=5 Participants
Race/Ethnicity, Customized Dagestani	1 Participants n=5 Participants
Race/Ethnicity, Customized Kazah	1 Participants n=5 Participants
Race/Ethnicity, Customized Uzbek	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: Included participants that had data for at least one post-baseline assessment of any efficacy measurement

FVC is a standard pulmonary function test. FVC is defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Baseline FVC will be the average of the highest FVC measurement recorded at the Screening and Day 1. The FVC at Week 26 will be the average of the highest FVC measurement recorded on two separate days at Week 26.

Outcome measures

Outcome measures
Measure	Pirfenidone n=55 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Change From Baseline to Week 26 in Absolute Millilitre (mL) Forced Vital Capacity (FVC)	128.78 Milliliter (mL) Interval -26.84 to 284.4

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: Included participants that had data for at least one post-baseline assessment of any efficacy measurement

Predicted FVC is based on sex, age, and height of a person. Percent predicted FVC (in %) = \[(observed FVC)/(predicted FVC)\]\*100.

Outcome measures

Outcome measures
Measure	Pirfenidone n=55 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Change From Baseline to Week 26 in Percent (%) Predicted FVC	-0.10 percent predicted Interval -3.18 to 2.99

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: Included participants that had data for at least one post-baseline assessment of any efficacy measurement and for the 6MWT at Week 26

Baseline 6MWT distance will be the average of the measurements recorded at the Screening and Day 1 visits. The 6MWT distance at Week 26 will be defined as the average of the 6MWT distance recorded on two separate days at Week 26.

Outcome measures

Outcome measures
Measure	Pirfenidone n=49 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Change From Baseline to Week 26 in 6-Minute Walk Test (6MWT) Distance Decline of >= 50 m	14 Participants
Change From Baseline to Week 26 in 6-Minute Walk Test (6MWT) Distance Decline of <50 m to 0 m	11 Participants
Change From Baseline to Week 26 in 6-Minute Walk Test (6MWT) Distance Improvement of >= 0 m	24 Participants

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: Included participants that had data for at least one post-baseline assessment of any efficacy measurement

The EQ-5D-5L is a self-reported health status questionnaire that consists of six questions used to calculate a health utility score for use in health economic analysis. There are two components to the EQ-5D-5L: a five-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Overall scores range from 0 to 1, with low scores representing a higher level of dysfunction.

Outcome measures

Outcome measures
Measure	Pirfenidone n=55 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Change From Baseline to Week 26 in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score	-0.0288 score on scale Standard Deviation 0.1820

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: Included participants that had data for at least one post-baseline assessment of any efficacy measurement

The EQ-5D-5L is a self-reported health status questionnaire that consists of six questions used to calculate a health utility score for use in health economic analysis. There are two components to the EQ-5D-5L: a five-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.

Outcome measures

Outcome measures
Measure	Pirfenidone n=55 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Change From Baseline to Week 26 in EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score	-0.6 score on scale Standard Deviation 17.16

SECONDARY outcome

Timeframe: Up to Week 52

An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (eg, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A treatment-emergent adverse event (TEAE) is defined as an adverse event with an onset that occurs after receiving study drug. Serious adverse event is any untoward medical occurrence at any dose that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of hospitalization, or resulted in persistent or significant disability/incapacity or congenital anomaly/birth defect.

Outcome measures

Outcome measures
Measure	Pirfenidone n=60 Participants Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) TEAEs	81.7 percentage of participants
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) TESAEs	16.7 percentage of participants

Adverse Events

Pirfenidone

Serious events: 10 serious events

Other events: 32 other events

Deaths: 7 deaths

Serious adverse events

Serious adverse events
Measure	Pirfenidone n=60 participants at risk Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Respiratory, thoracic and mediastinal disorders Idiopathic pulmonary fibrosis	3.3% 2/60 • Number of events 3 • Up to 52 Weeks
Respiratory, thoracic and mediastinal disorders Bronchitis chronic	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
General disorders Death	3.3% 2/60 • Number of events 2 • Up to 52 Weeks
General disorders Sudden cardiac death	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Cardiac disorders Atrial fibrillation	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Cardiac disorders Atrial flutter	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Infections and infestations Bronchitis bacterial	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Infections and infestations Cholecystitis infective	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Infections and infestations Pneumonia	1.7% 1/60 • Number of events 1 • Up to 52 Weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Adenocarcinoma of colon	1.7% 1/60 • Number of events 1 • Up to 52 Weeks

Other adverse events

Other adverse events
Measure	Pirfenidone n=60 participants at risk Participants administered pirfenidone 2403 milligram per day (mg/d) orally for 26 weeks
Gastrointestinal disorders Nausea	26.7% 16/60 • Up to 52 Weeks
Gastrointestinal disorders Dyspepsia	13.3% 8/60 • Up to 52 Weeks
Gastrointestinal disorders Vomiting	13.3% 8/60 • Up to 52 Weeks
Gastrointestinal disorders Diarrhoea	11.7% 7/60 • Up to 52 Weeks
Metabolism and nutrition disorders Decreased appetite	21.7% 13/60 • Up to 52 Weeks
Respiratory, thoracic and mediastinal disorders Dyspnoea	10.0% 6/60 • Up to 52 Weeks
Respiratory, thoracic and mediastinal disorders Cough	6.7% 4/60 • Up to 52 Weeks
Investigations Weight decreased	6.7% 4/60 • Up to 52 Weeks
Skin and subcutaneous tissue disorders Pruritus	6.7% 4/60 • Up to 52 Weeks

Additional Information

Medical Communications

Hoffmann-La Roche

Phone: 800 821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights
Publication restrictions are in place

Restriction type: OTHER