Pilot Study of Pirfenidone in Pulmonary Fibrosis With Anti-myeloperoxydase Antibodies

NCT03385668 | Completed Phase 2

• 2 other identifiers: P161001J2017-002782-22
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine wether pirfenidone is safe and effective in the treatment of pulmonary fibrosis with anti-myeloperoxydase (MPO) antibodies or pulmonary fibrosis with anti-MPO associated vasculitis.

Conditions

Pulmonary Fibrosis

Keywords

Pulmonary fibrosis; Antibodies, Antineutrophil Cytoplasmic; Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis; Pirfenidone; Microscopic Polyangiitis; Interstitial lung disease

Outcome Measures

Primary Outcomes (1)

• Treatment efficacy measured by the absolute change in percent predicted forced vital capacity (%FVC)

  Treatment efficacy at Week 52 measured by the absolute change from baseline to Week 52 in percent predicted forced vital capacity (%FVC) : * Patients with progressive disease will be defined as absolute decline of 10% or more in %FVC. Missing values or death will be also considered as progressive. * Patients with non-progressive disease will be defined as improvement or no decline in %FVC or a decline of %FVC\<10%.

  52 weeks

Secondary Outcomes (15)

• Adverse Events (AE)

  56 weeks corresponding to 28 days after the last dose of study drug

• Treatment efficacy measured by the absolute change in percent predicted forced vital capacity (%FVC)

  24 weeks

• Relative change in in percent predicted forced vital capacity

  52 weeks

• Absolute change in in percent predicted forced vital capacity

  52 weeks

• Relative change in in percent predicted forced vital capacity

  24 weeks

Study Arms (1)

Pirfenidone

EXPERIMENTAL

All patients will receive Pirfenidone

Drug: Pirfenidone

Interventions

Pirfenidone DRUG

Pirfenidone at a dose of 2403 mg/day for 50 weeks, after a 2 weeks period of titration (801 mg/day for one week then 1602 mg/day for one week).

Pirfenidone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \> 18 years
• Definite or possible Usual Interstitial Pneumonia or Non Specific Interstitial Pneumonia based on high-resolution computed tomography
• Presence of pulmonary fibrosis, defined as a range of 50 to 90% of the %FVC and a range of 30 to 90% of the %DLCO
• Pulmonary fibrosis refractory (according to the investigator's judgment) to a conventional regimen used for anti-MPO associated vasculitis when a treatment against vasculitis has been used
• Have the ability to understand the requirements of the study, provide written informed consent (including consent for the use and disclosure of research-related health information) and comply with the study protocol procedures (including required study visits)
• Have affiliation with a mode of social security (profit our being entitled).

You may not qualify if:

• Other type of systemic vasculitis;
• Active vasculitis defined by Birmingham Vasculitis Activity Score \>3 (BVAS) ;
• Contraindication to Pirfenidone;
• Unable to perform pulmonary function test (PFT);
• Pregnancy or lactation. Women of childbearing capacity are required to have a negative serum pregnancy test before treatment and must agree to maintain highly effective contraception by practicing abstinence or by using an effective method of birth control from the date of consent through the end of the study : implants of levonorgestrel; injectable progesterone; any intrauterine device (IUD) with a documented failure rate of less than 1% per year; oral contraceptives (either combined or progesterone only); double barrier method (condom, cervical cap or diaphragm with spermicidal agent); transdermal contraceptive patch; male partner who is sterile prior to the female subject's entry into the study and is the sole sexual partner for the female subject;
• Any of the following liver function test criteria above specified limits: total bilirubin above 1,5 times the upper limit of normal (ULN), excluding patients with Gilbert's syndrome; aspartate (AST)/Glutamate Oxaloacétique Transaminase (SGOT) or alanine aminotransferase (ALT)/Glutamate Pyruvate Transaminase (SGPT), (AST/SGOT or ALT/SGPT) \>3 × ULN; alkaline phosphatase \>2.5 × ULN;
• Creatinine clearance (CrCl\<30) mL/min, calculated using the Cockcroft-Gault formula at screening
• Current treatment with Nintedanib or past treatment with Nintedanib in the last 12 months;
• Current treatment with Fluvoxamine or past treatment with Fluvoxamine in the last 28 days before screening
• Prior use of Pirfenidone or known hypersensitivity to any of the components of study treatment;
• Expected to receive a lung transplant within 1 year from randomization or, on a lung transplant waiting list at randomization;
• Associated connective tissue disease (such as systemic sclerosis).;
• Electrocardiogram (ECG), with a heart-rate-corrected QT interval (corrected using Fridericia's formula, QTcF) ≥ 500 ms at Screening, or a family or personal history of long QT syndrome;
• Treatment with Cyclophosphamide in the last 3 months;
• Current smoking or past smoking in the last 3 months.

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Roche Pharma AG: collaborator
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (1)

Cochin Hospital

Paris, 75014, France

Location

Related Publications (3)

• Hervier B, Pagnoux C, Agard C, Haroche J, Amoura Z, Guillevin L, Hamidou MA; French Vasculitis Study Group. Pulmonary fibrosis associated with ANCA-positive vasculitides. Retrospective study of 12 cases and review of the literature. Ann Rheum Dis. 2009 Mar;68(3):404-7. doi: 10.1136/ard.2008.096131. Epub 2008 Oct 28.

  PMID: 18957485 BACKGROUND

• King TE Jr, Bradford WZ, Castro-Bernardini S, Fagan EA, Glaspole I, Glassberg MK, Gorina E, Hopkins PM, Kardatzke D, Lancaster L, Lederer DJ, Nathan SD, Pereira CA, Sahn SA, Sussman R, Swigris JJ, Noble PW; ASCEND Study Group. A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. N Engl J Med. 2014 May 29;370(22):2083-92. doi: 10.1056/NEJMoa1402582. Epub 2014 May 18.

  PMID: 24836312 BACKGROUND

• Lancaster L, Albera C, Bradford WZ, Costabel U, du Bois RM, Fagan EA, Fishman RS, Glaspole I, Glassberg MK, King TE Jr, Lederer DJ, Lin Z, Nathan SD, Pereira CA, Swigris JJ, Valeyre D, Noble PW. Safety of pirfenidone in patients with idiopathic pulmonary fibrosis: integrated analysis of cumulative data from 5 clinical trials. BMJ Open Respir Res. 2016 Jan 12;3(1):e000105. doi: 10.1136/bmjresp-2015-000105. eCollection 2016.

  PMID: 26835133 BACKGROUND

MeSH Terms

Conditions

Pulmonary Fibrosis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Microscopic Polyangiitis; Lung Diseases, Interstitial

Interventions

pirfenidone

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Jonathan London, MD

  Cochin Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 7, 2017
• First Posted: December 28, 2017
• Study Start: January 31, 2018
• Primary Completion: July 24, 2020
• Study Completion: July 24, 2020
• Last Updated: November 20, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)