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A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis

Purpose

A randomized, double-blind, placebo-controlled clinical study to evaluate the safety and efficacy of 2 doses of inhaled pirfenidone (AP01) versus placebo on top of standard of care in participants with PPF over 52 weeks.

Condition

Progressive Pulmonary Fibrosis

Eligibility

Eligible Ages: Over 18 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

Inclusion Criteria

Participant meets criteria for PPF, as follows: - In subjects with interstitial lung disease (ILD) of known or unknown etiology other than idiopathic pulmonary fibrosis (IPF) who have radiological evidence of pulmonary fibrosis, PPF is defined as: Physiological evidence of disease progression with at least 1 of the following criteria despite treatment with approved or unapproved medications commonly used in practice (per Investigator): 1. Relative decline in FVC ≥10% predicted within the previous 24 months based on documented historical spirometry assessments 2. Relative decline in FVC ≥5% to <10% predicted within the previous 24 months based on documented historical spirometry assessments with at least 1 of the 2 following criteria: - Worsening respiratory symptoms (Note: Changes attributable to comorbidities e.g., infection, heart failure must be excluded) OR - Radiological (HRCT) evidence of disease progression per a local or central radiologist (from historical HRCT taken up to 24 months prior to Screening Visit 1), for example: - Increased extent or severity of traction bronchiectasis and bronchiolectasis - New ground-glass opacity with traction bronchiectasis - New fine reticulation - Increased extent or increased coarseness of reticular abnormality - New or increased honeycombing - Increased lobar volume loss 3. Worsening of respiratory symptoms (Note: Changes attributable to comorbidities e.g., infection, heart failure must be excluded) AND radiological (HRCT) evidence of disease progression per a local or central radiologist - Meeting all of the following criteria during the Screening Period: a. FVC ≥45% of predicted normal at Screening Visit 1, b. Forced expiratory volume at 1 second (FEV1)/FVC ≥0.7 or ≥age-adjusted lower limit of normal at Screening Visit 1, c. Diffusing capacity of lung for carbon monoxide (DLCO) ≥30% of predicted, corrected for hemoglobin at Screening Visit 1, d. Acceptability: Participants can perform acceptable spirometry (i.e., meet American Thoracic Society (ATS)/ European Respiratory Society (ERS) acceptability criteria at both Screening Visits). • For subjects already on nintedanib (up to 30% of subjects): Must have been on nintedanib for at least 6 months prior to Screening with or without dose adjustments and/or drug interruptions during that period. For subjects who have discontinued nintedanib prior to Screening: Must have been off of nintedanib for a minimum of 12 weeks.

Exclusion Criteria

Current treatment with oral pirfenidone or treatment with oral pirfenidone within 3 months prior to Screening. - Elevated liver enzymes and liver injury at Screening defined as: 1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ˃ 3 times the upper limit of normal (ULN) 2. Bilirubin >2.0 x ULN - Renal disease with a creatinine clearance < 30 mL/min, calculated according to the Chronic Kidney Disease Epidemiology Collaboration formula. Retesting is allowed once. - Diagnosis of idiopathic pulmonary fibrosis (IPF) based on the ATS diagnostic algorithm for IPF. UIP that is not idiopathic, for example related to rheumatoid arthritis (RA), familial interstitial lung disease (ILD), or other is not exclusionary. - Greater extent of emphysema than of fibrotic ILD on HRCT. Note: CT results must be confirmed through the central over read process. - Significant clinical worsening of PPF between Screening - Participants who cannot meet protocol-specified Baseline stability criteria. FVC Baseline stability is defined as the FVC assessments at Visit 3 being within ±12% of the mean of the FVC assessments obtained at the 2 preceding visits. At Visit 3, if the pre-dose FVC is outside of ±12% range, the participant will not be randomized and will be considered a screen failure.

Study Design

Phase: Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

Arm	Description	Assigned Intervention
Experimental AP01 High Dose BID	Pirfenidone Solution for Inhalation	Drug: AP01 Oral inhalation solution Other names: Pirfenidone Solution
Experimental AP01 Low Dose BID	Pirfenidone Solution for Inhalation	Drug: AP01 Oral inhalation solution Other names: Pirfenidone Solution
Placebo Comparator Placebo BID	Placebo solution for inhalation	Other: Placebo Placebo oral inhalation solution

Recruiting Locations

University of Alabama at Birmingham
Birmingham 4049979, Alabama 4829764 35205

Pulmonary Associates, PA
Phoenix 5308655, Arizona 5551752 85032

University of Southern California
Los Angeles 5368361, California 5332921 90033

Cedars-Sinai
Los Angeles 5368361, California 5332921 90048

UCLA
Los Angeles 5368361, California 5332921 90095

Newport Native MD, Inc.
Newport Beach 5376890, California 5332921 92663

Paradigm Clinical Research - Redding
Redding 5570160, California 5332921 96001

University of Colorado, Anschutz Medical Campus
Aurora 5412347, Colorado 5417618 80045

National Jewish Health
Denver 5419384, Colorado 5417618 80206

UCONN Health
Farmington 4834272, Connecticut 4831725 06030

Yale University
New Haven 4839366, Connecticut 4831725 06519

Clinical Site Partners, LCC
Leesburg 4161771, Florida 4155751 34748

Renstar Medical Research
Ocala 4166673, Florida 4155751 34470

Central Florida Pulmonary Group
Orlando 4167147, Florida 4155751 32803

SEC Clinical Research
Pensacola 4168228, Florida 4155751 32503

Clinical Site Partners
Winter Park 4178560, Florida 4155751 32789

Piedmont Healthcare, Inc.
Atlanta 4180439, Georgia 4197000 30309

Northwestern University
Chicago 4887398, Illinois 4896861 60611

Endeavor Health
Evanston 4891382, Illinois 4896861 60201

Loyola University Medical Center
Maywood 4901514, Illinois 4896861 60153

The University of Kansas Medical Center
Kansas City 4273837, Kansas 4273857 66160

University of Maryland
Baltimore 4347778, Maryland 4361885 21201

Johns Hopkins University
Baltimore 4347778, Maryland 4361885 21224

Beth Israel Deaconess Medical Center
Boston 4930956, Massachusetts 6254926 02215

University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109

University of Minnesota
Minneapolis 5037649, Minnesota 5037779 55455

Mayo Clinic Rochester
Rochester 5043473, Minnesota 5037779 55905

Hannibal Regional Healthcare System
Hannibal 4389418, Missouri 4398678 63401

Northwell Health - Mount Kisco
Mount Kisco 5127744, New York 5128638 10549

NYU Langone Health
New York 5128581, New York 5128638 10017

Weill Cornell
New York 5128581, New York 5128638 10021

Icahn School of Medicine at Mount Sinai
New York 5128581, New York 5128638 10029

Columbia University
New York 5128581, New York 5128638 10032

Montefiore Medical Center
The Bronx 5110266, New York 5128638 10467

Duke University
Durham 4464368, North Carolina 4482348 27710

Pulmonix
Greensboro 4469146, North Carolina 4482348 27403

Piedmont HealthCare, PA
Statesville 4493316, North Carolina 4482348 28625

Accellacare
Wilmington 4499379, North Carolina 4482348 28401

Southeastern Research Center
Winston-Salem 4499612, North Carolina 4482348 27103

University of Cincinnati
Cincinnati 4508722, Ohio 5165418 45267

Cleveland Clinic
Cleveland 5150529, Ohio 5165418 44195

Summit Health
Bend 5713587, Oregon 5744337 97701

The Oregon Clinic Pulmonary East
Portland 5746545, Oregon 5744337 97220

The Oregon Clinic Pulmonary West
Portland 5746545, Oregon 5744337 97225

The Pennsylvania State University
Hershey 5193342, Pennsylvania 6254927 17033

Thomas Jefferson University
Philadelphia 4560349, Pennsylvania 6254927 19107

Temple University
Philadelphia 4560349, Pennsylvania 6254927 19140

Lowcountry Lung and Critical Care
Charleston 4574324, South Carolina 4597040 29406

Medical University of South Carolina
Charleston 4574324, South Carolina 4597040 29425

Clinical Trials Center of Middle Tennessee
Franklin 4623560, Tennessee 4662168 37067

Vanderbilt Lung Institute
Nashville 4644585, Tennessee 4662168 37204

Baylor Scott & White Research Institute, Baylor University Medical Center
Dallas 4684888, Texas 4736286 75246

El Paso Pulmonary Association
El Paso 5520993, Texas 4736286 79902

Baylor College of Medicine
Houston 4699066, Texas 4736286 77030

The University of Texas Health Science Center
Houston 4699066, Texas 4736286 77030

Metroplex Pulmonary and Sleep Center, PA
McKinney 4710178, Texas 4736286 75069

Intermountain Medical Center
Murray 5778755, Utah 5549030 84157

University of Utah Health
Salt Lake City 5780993, Utah 5549030 84108

Inova Healthcare
Falls Church 4758390, Virginia 6254928 22042

University of Washington
Seattle 5809844, Washington 5815135 98195

More Details

NCT ID: NCT06329401
Status: Recruiting
Sponsor: Avalyn Pharma Inc.

Study Contact

Craig S. Conoscenti, MD
206-707-0304
cconoscenti@avalynpharma.com

Detailed Description

This is a randomized, double-blind, placebo-controlled clinical study to evaluate the safety and efficacy of 2 doses of AP01 (pirfenidone solution for inhalation) versus placebo on top of standard of care in participants with PPF over 52 weeks. Up to 300 eligible participants will be randomized to 1 of 3 treatment arms: AP01 high dose, AP01 low dose, or placebo.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.