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About
This study is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ensifentrine inhalation suspension (3 mg) delivered twice daily via standard jet nebulizer over at least 24 weeks, compared to placebo, in subjects with non-cystic fibrosis bronchiectasis (NCFBE).
Full description
The primary objective of this study is to assess the effect of ensifentrine vs placebo in addition to standard of care on pulmonary exacerbations, symptoms and quality of life in participants with NCFBE. The study is designed as a pulmonary exacerbation event-driven study where participants will be treated for ≥ 24 weeks and until at least 120 subjects have experienced at least 1 protocol-defined pulmonary exacerbation. Participants will be randomized to receive either ensifentrine suspension or placebo via standard jet nebulizer during the treatment period and neither participants nor study staff will know which a participant is receiving.
Enrollment
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Volunteers
Inclusion criteria
Males are eligible to participate if they agree to use contraception as described in the contraceptive guidance from Screening and throughout the study and for at least 30 days after the last dose of blinded study medication
Females are eligible to participate if they are not pregnant, not breastfeeding, and 1 of the following conditions apply:
Clinical history consistent with bronchiectasis (cough, chronic sputum production, and/or recurrent respiratory infections) confirmed by chest CT demonstrating bronchiectasis affecting 1 or more lobes. Confirmation may be based on prior chest CT within the prior 5 years; subjects whose past CT image records are not available will require chest CT scan during screening Notes: If a subject has no clinical history consistent with bronchiectasis, they may not be re-screened
Current sputum producer with a history of chronic expectoration and able to provide sputum sample spontaneously at the clinic during screening
≥ 1 documented pulmonary exacerbation defined by an antibiotic prescription by a physician for the signs and symptoms of respiratory infections in the past 12 months before screening
Capable of using the study nebulizer correctly
Ability to perform acceptable spirometry in accordance with American Thoracic Society and European Respiratory Society guidelines
Exclusion criteria
A diagnosis of COPD or a primary diagnosis of asthma, as judged by the investigator
Bronchiectasis due to cystic fibrosis, hypogammaglobulinemia common variable immunodeficiency, severe immunodeficiency, or requirement for treatment with intravenous immunoglobulin
Current smoker defined as by the Centers for Disease Control and Prevention (CDC)
Former cigarette smokers with a history of cigarette smoking ≥ 10 pack years at Screening [number of pack years = (number of cigarettes per day / 20) × number of years smoked (e.g., 20 cigarettes per day for 10 years, or 10 cigarettes per day for 20 years)]. Pipe and/or cigar use cannot be used to calculate pack-year history. Former smokers are defined as those who have stopped smoking for at least 6 months prior to Screening
A diagnosis of primary ciliary dyskinesia
Current treatment for nontuberculous mycobacterial lung infection, allergic bronchopulmonary aspergillosis, or tuberculosis
Presence of acute exacerbation or acute infection that required acute treatment within 28 days of randomization
Use of the following prohibited medications within the designated time periods:
Initiated or altered therapy within 90 days of randomization with:
Initiated or altered therapy with ICS within 4 weeks prior to randomization
Unable to withhold short-acting beta-agonists or short-acting muscarinic antagonists for ≥ 4 hours prior to spirometry
Significant hemoptysis (≥ 300 mL or requiring blood transfusion) within 6 weeks prior to randomization
Currently participating in or scheduled to participate in an intensive pulmonary rehabilitation program (a maintenance rehabilitation program is allowed if their schedule and procedure will be consistent for the duration of the study)
Current or chronic history of unstable liver disease defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices or persistent jaundice, cirrhosis, or known hepatic or biliary abnormalities except for a diagnosis of Gilbert syndrome or asymptomatic gallstones Note: Chronic stable hepatitis B and C is not exclusionary if the subject otherwise meets study entry criteria
History of or current malignancy of any organ system, treated or untreated within the past 5 years, except for localized basal or squamous cell carcinoma of the skin
Estimated glomerular filtration rate (eGFR) < 30 mL/min
Alanine aminotransferase (ALT) ≥ 2 × upper limit of normal (ULN), aspartate aminotransferase (AST) ≥ 2 × ULN, alkaline phosphatase and/or bilirubin > 1.5 × ULN (isolated bilirubin > 1.5 × ULN is acceptable only in subjects with a diagnosis of Gilbert's syndrome)
Participation in any other interventional, clinical studies (drugs or devices) within 30 days before Day 1, or 5 half-lives, whichever is longer
Intolerance of or hypersensitivity to ensifentrine or any of its excipients/components
Current or history of drug or alcohol abuse within the past 5 years
Significantly abnormal ECG finding
Primary purpose
Allocation
Interventional model
Masking
180 participants in 2 patient groups, including a placebo group
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Central trial contact
Verona Pharma Clinical Trials
Data sourced from clinicaltrials.gov
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