Effect of Ensifentrine Treatment on CAT Score

NCT06460493 | Completed Phase 3 Results FDA Drug

• 1 other identifier: RPL554-CO-303
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this open-label, single center trial is to assess the effect of twice daily nebulized ensifentrine (3 mg) on COPD symptoms measured by the COPD Assessment Test (CAT™) scores over 12 weeks in subjects with symptomatic, moderate to severe COPD, taking maintenance LAMA/LABA or LAMA/LABA/ICS therapy. Subjects will continue the maintenance treatment during study participation.

Conditions

COPD

Outcome Measures

Primary Outcomes (1)

• The Proportion of CAT Score Responders in the Full Analysis Set at Week 12

  Responders are defined as an improvement (decrease) from baseline of ≥2 points. The COPD Assessment Test(tm) (CAT) is a one-page, 8-item questionnaire suitable for completion by patients diagnosed with COPD. When completing the questionnaire, individuals rate their experience on a 6-point scale, ranging from 0 (no impairment) to 5 (maximum impairment) with a scoring range of 0-40. Higher scores indicate greater disease impact. The individual items are: Cough, Mucus, Chest Tight, Breathless, Limited Activities, Confident, Sleep and Energy). CAT was self-completed at baseline, week 6 and week 12.

  12 Weeks

Secondary Outcomes (10)

• The Proportion of CAT Score Responders in the Full Analysis Set at Week 6

  6 Weeks

• Mean Change From Baseline - CAT Score in the Full Analysis Set

  At week 6 and week 12

• Mean Change From Baseline - CAT Individual Item (1, Cough) in the Full Analysis Set

  At week 6 and week 12

• Mean Change From Baseline - CAT Individual Item (2, Mucus) in the Full Analysis Set

  At week 6 and week 12

• Mean Change From Baseline - CAT Individual Item (3, Chest Tight) in the Full Analysis Set

  At week 6 and week 12

Other Outcomes (2)

• Mean Change From Baseline in Blood Pressure in the Safety Analysis Set

  12 Weeks

• Mean Change From Baseline in Pulse Rate in the Safety Analysis Set

  12 Weeks

Study Arms (1)

Ensifentrine

EXPERIMENTAL

Inhaled ensifentrine twice daily for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) COPD therapy.

Drug: Ensifentrine 3 mg twice daily

Interventions

Ensifentrine 3 mg twice daily DRUG

All subjects will receive ensifentrine.

Also known as: RPL554

Ensifentrine

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of giving informed consent.
• Current or former cigarette smokers with a history of cigarette smoking ≥ 10 pack years.
• Established clinical history of moderate to severe COPD.
• Pre- and post-albuterol FEV1/FVC ratio of \< 0.70.
• Post-albuterol FEV1 ≥ 30 % and ≤ 75% of predicted normal.
• ≥2 on the modified Medical Research Council (mMRC) Dyspnea Scale.
• ≥10 on the COPD Assessment Test™.
• Taking stable dual (LAMA+LABA) or triple (LAMA+LABA+ICS) maintenance therapy for at least 8 weeks.
• Capable of using a standard jet nebulizer and performed spirometry assessment.

You may not qualify if:

• Hospitalizations for COPD (or COPD exacerbation), pneumonia, or other serious infection or treatment with oral or parenteral (oral, intravenous, or intramuscular) glucocorticoids within the past 12 weeks.
• Lower respiratory tract infection within the past 6 weeks or an active infection.
• History of life-threatening COPD, including Intensive Care Unit admission and/or requiring intubation within 1-year.
• Major surgery (requiring general anesthesia) within the past 6 weeks, lack of full recovery from surgery, or planned surgery through the end of the study.
• Concomitant clinically significant pulmonary disease other than COPD (e.g., asthma, tuberculosis, lung cancer, bronchiectasis, sarcoidosis, lung fibrosis, interstitial lung diseases, sleep apnea (unless controlled with stable continuous positive airway pressure \[CPAP\] use), known alpha-1 antitrypsin deficiency, core pulmonale or other non-specific pulmonary disease).
• Severe comorbidities including unstable cardiac, (e.g., myocardial infarction within 1 year prior to screening, unstable angina within 6 months, or unstable or life-threatening arrhythmia requiring intervention within 3 months) or any other clinically significant medical conditions including uncontrolled diseases (e.g., endocrine, neurological, hepatic, gastrointestinal, renal, hematological, urological, immunological, psychiatric or ophthalmic diseases) that would, in the opinion of the Investigator, preclude the subject from safely completing the required tests or the study, or is likely to result in disease progression that would require withdrawal of the subject.
• HIV infection or other immunodeficiency.
• Previous lung resection or lung reduction surgery within 1-year.
• Long term oxygen use defined as oxygen therapy prescribed for greater than 12 hours per day.
• Pulmonary rehabilitation unless such treatment has been stable for at least 4 weeks and remains stable during the study.
• 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.
• Significant psychiatric disease that would likely result in the subject not being able to complete the study, in the opinion of the Investigator.
• Findings on physical examination that an investigator considers to be clinically significant.
• Known alanine aminotransferase (ALT) ≥ 2 × upper limit of normal (ULN), alkaline phosphatase and/or bilirubin \> 1.5 × ULN (isolated bilirubin \> 1.5 × ULN is acceptable if fractionated bilirubin \< 35%).
• Known diagnosis of severe chronic kidney disease.

Sponsors & Collaborators

• Verona Pharma plc: lead
• Midwest Chest Consultants: collaborator

Study Sites (1)

Midwest Chest Consultants

Saint Charles, Missouri, 63301, United States

Location

MeSH Terms

Conditions

Pulmonary Disease, Chronic Obstructive

Interventions

ensifentrine

Condition Hierarchy (Ancestors)

Lung Diseases, Obstructive; Lung Diseases; Respiratory Tract Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Clinical Development
• Organization: Verona Pharma

clinicaltrials@veronapharma.com

844-341-9901

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 10, 2024
• First Posted: June 14, 2024
• Study Start: June 11, 2024
• Primary Completion: November 26, 2024
• Study Completion: December 3, 2024
• Last Updated: April 18, 2025
• Results First Posted: April 18, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)