Active, not recruiting
Phase 2
A Study Evaluating the Safety and Efficacy of Inhaled AP01 in Participants With Progressive Pulmonary Fibrosis
Condition(s) studied
Pulmonary FibrosisProgressive Pulmonary FibrosisPulmonary Fibrosis Secondary to Systemic SclerosisPulmonary Fibrosis, Interstitial Lung DiseaseInterstitial Lung DiseaseInterstitial Lung Disease Due to Connective Tissue Disease (Disorder)Interstitial Lung Disease in Patients With Rheumatoid ArthritisInterstitial Lung Disease With Progressive Fibrotic Phenotype in Diseases Classified ElsewhereHypersensitivity PneumonitisInterstitial Lung Disease With Systemic Sclerosis
Investigational drug(s) / intervention(s)
AP01: Oral inhalation solution
Placebo: Placebo oral inhalation solution
Study summary
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.
Eligibility
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 background therapy (nintedanib or nerandomilast 18 mg twice daily \[up to 30% of subjects\]): Must have been on background therapy for at least 12 weeks prior to Screening with or without dose adjustments and/or drug interruptions during that period. For subjects who have discontinued background therapy prior to Screening: Must have been off for a minimum of 12 weeks.
Exclusion Criteria:
* Current treatment with oral pirfenidone or nerandomilast 9 mg twice daily dose 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 Visit 1 and Visit 3
* 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.
Primary outcome measure(s)
- To evaluate the effect of AP01 high dose twice a day (BID) or AP01 low dose twice a day (BID) compared to placebo twice a day (BID) — Week 52
Change from baseline in forced vital capacity (FVC) (mL)
Trial sites (154)
| Facility | City | Region | Status |
| University of Alabama at Birmingham |
Birmingham |
Alabama |
|
| Mayo Clinic- Scottsdale |
Scottsdale |
Arizona |
|
| University of Southern California |
Los Angeles |
California |
|
| Cedars-Sinai |
Los Angeles |
California |
|
| UCLA |
Los Angeles |
California |
|
| Newport Native MD, Inc. |
Newport Beach |
California |
|
| Paradigm Clinical Research - Redding |
Redding |
California |
|
| University of California - San Francisco |
San Francisco |
California |
|
| University of Colorado, Anschutz Medical Campus |
Aurora |
Colorado |
|
| National Jewish Health |
Denver |
Colorado |
|
| UCONN Health |
Farmington |
Connecticut |
|
| Yale University |
New Haven |
Connecticut |
|
| Clinical Site Partners, LCC |
Leesburg |
Florida |
|
| Renstar Medical Research |
Ocala |
Florida |
|
| Clinical Site Partners |
Winter Park |
Florida |
|
| Piedmont Healthcare, Inc. |
Atlanta |
Georgia |
|
| Northwestern University |
Chicago |
Illinois |
|
| Endeavor Health |
Evanston |
Illinois |
|
| Loyola University Medical Center |
Maywood |
Illinois |
|
| The University of Kansas Medical Center |
Kansas City |
Kansas |
|
| University of Maryland |
Baltimore |
Maryland |
|
| Johns Hopkins University |
Baltimore |
Maryland |
|
| Beth Israel Deaconess Medical Center |
Boston |
Massachusetts |
|
| University of Michigan |
Ann Arbor |
Michigan |
|
| University of Minnesota |
Minneapolis |
Minnesota |
|
| Mayo Clinic Rochester |
Rochester |
Minnesota |
|
| Hannibal Regional Healthcare System |
Hannibal |
Missouri |
|
| Northern Westchester Hospital |
Mount Kisco |
New York |
|
| Northwell Health - Mount Kisco |
Mount Kisco |
New York |
|
| NYU Langone Health |
New York |
New York |
|
| Weill Cornell |
New York |
New York |
|
| Icahn School of Medicine at Mount Sinai |
New York |
New York |
|
| Columbia University |
New York |
New York |
|
| Montefiore Medical Center |
The Bronx |
New York |
|
| Duke University |
Durham |
North Carolina |
|
| Piedmont HealthCare, PA |
Statesville |
North Carolina |
|
| Accellacare |
Wilmington |
North Carolina |
|
| Southeastern Research Center |
Winston-Salem |
North Carolina |
|
| University of Cincinnati |
Cincinnati |
Ohio |
|
| Cleveland Clinic |
Cleveland |
Ohio |
|
+ 114 more sites — see the full list on the official registry below.
More Avalyn Pharma Inc. trials in the UK
Other trials for the same condition