NCT03208933

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

Completed Phase 3 Results posted Last updated 20 November 2020

What this trial tests

Phase 3 trial testing Pirfenidone in Idiopathic Pulmonary Fibrosis in 60 participants. Completed in 13 November 2019.

Timeline

23 October 2017

Primary endpoint
13 November 2019

13 November 2019

Quick facts

Lead sponsor	Hoffmann-La Roche
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	60
Start date	23 October 2017
Primary completion	13 November 2019
Estimated completion	13 November 2019
Sites	11 locations across Russia

Drugs / interventions tested

• Pirfenidone — full drug profile →

Conditions studied

• Idiopathic Pulmonary Fibrosis — all drugs for Idiopathic Pulmonary Fibrosis →

Sponsor

Hoffmann-La Roche — full company profile →

Who can join

Adults 40 to 80, any sex, with Idiopathic Pulmonary Fibrosis. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change From Baseline to Week 26 in Absolute Millilitre (mL) Forced Vital Capacity (FVC) Primary · Baseline, Week 26

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.

Group	Value	95% CI
Pirfenidone	128.78	-26.84 – 284.40

Change From Baseline to Week 26 in Percent (%) Predicted FVC Primary · Baseline, Week 26

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

Group	Value	95% CI
Pirfenidone	-0.10	-3.18 – 2.99

Change From Baseline to Week 26 in 6-Minute Walk Test (6MWT) Distance Secondary · Baseline, 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.

Decline of >= 50 m

Group	Value	95% CI
Pirfenidone	14

Decline of <50 m to 0 m

Group	Value	95% CI
Pirfenidone	11

Improvement of >= 0 m

Group	Value	95% CI
Pirfenidone	24

Change From Baseline to Week 26 in EuroQol 5-Dimension 5-Level (EQ-5D-5L) Questionnaire Index Score Secondary · Baseline, Week 26

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.

Group	Value	95% CI
Pirfenidone	-0.0288	± 0.1820

Change From Baseline to Week 26 in EQ-5D-5L Visual Analogue Scale (EQ-5D-5L VAS) Score Secondary · Baseline, Week 26

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 r

Group	Value	95% CI
Pirfenidone	-0.6	± 17.16

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs) Secondary · 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 medi

TEAEs

Group	Value	95% CI
Pirfenidone	81.7

TESAEs

Group	Value	95% CI
Pirfenidone	16.7

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 52 Weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Pirfenidone

Serious: 10/60 (17%)

Deaths: 7/60

Serious adverse events (11 terms)

Reaction	System	Pirfenidone
Idiopathic pulmonary fibrosis	Respiratory, thoracic and mediastinal disorders	—
Death	General disorders	—
Bronchitis chronic	Respiratory, thoracic and mediastinal disorders	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—
Sudden cardiac death	General disorders	—
Atrial fibrillation	Cardiac disorders	—
Atrial flutter	Cardiac disorders	—
Bronchitis bacterial	Infections and infestations	—
Cholecystitis infective	Infections and infestations	—
Pneumonia	Infections and infestations	—
Adenocarcinoma of colon	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—

Other adverse events (9 terms — click to expand)

Reaction	System	Pirfenidone
Nausea	Gastrointestinal disorders	—
Decreased appetite	Metabolism and nutrition disorders	—
Dyspepsia	Gastrointestinal disorders	—
Vomiting	Gastrointestinal disorders	—
Diarrhoea	Gastrointestinal disorders	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Weight decreased	Investigations	—
Pruritus	Skin and subcutaneous tissue disorders	—

Most-reported serious reactions: Idiopathic pulmonary fibrosis, Death, Bronchitis chronic, Dyspnoea, Sudden cardiac death, Atrial fibrillation, Atrial flutter, Bronchitis bacterial.

Data from ClinicalTrials.gov NCT03208933 adverse events section.

Sponsor's own description

This study is a national, multicenter, interventional, non-randomized, non-controlled, open-label study to assess the effectiveness of pirfenidone in participants with IPF in Russian clinical practice.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Atypical p38 Signaling, Activation, and Implications for Disease.
   Burton JC, Antoniades W, Okalova J, Roos MM, et al · · 2021 · cited 52× · PMID 33920735 · DOI 10.3390/ijms22084183
2. Effects of Pirfenidone on Idiopathic Pulmonary Fibrosis Progression and Safety: Results of Multicenter Prospective Observational Study.
   Avdeev S, Ilkovich M, Terpigorev S, Moiseev S, et al · · 2023 · cited 4× · PMID 36836840 · DOI 10.3390/life13020483