Trial Outcomes & Findings for Effect of Ensifentrine Treatment on CAT Score (NCT NCT06460493)

NCT ID: NCT06460493

Last Updated: 2025-04-18

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 20 participants
• Primary outcome timeframe: 12 Weeks
• Results posted on: 2025-04-18

Participant Flow

Following the informed consent process, screening assessments were completed at visits 1 and 2.

If any inclusion criteria were not met during screening at visit 1 or 2, the subject was not treated with study medication. Study medication was dispensed and administered at visit 2 after all eligibility criteria were met.

Participant milestones

Measure	Ensifentrine 3 mg BID Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Overall Study STARTED	20
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Ensifentrine 3 mg BID Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Effect of Ensifentrine Treatment on CAT Score

Baseline characteristics by cohort

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Age, Continuous	73 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	18 Participants n=5 Participants
Mean baseline CAT score	18.3 units on a scale STANDARD_DEVIATION 6.4 • n=5 Participants

PRIMARY outcome

Timeframe: 12 Weeks

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

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.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
The Proportion of CAT Score Responders in the Full Analysis Set at Week 12	12 Participants

SECONDARY outcome

Timeframe: 6 Weeks

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

Responders are defined as an improvement (decrease) from baseline of ≥2 points. The COPD Assessment Test (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.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
The Proportion of CAT Score Responders in the Full Analysis Set at Week 6	8 Participants

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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. A decrease from baseline indicates an improvement in COPD symptoms.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Score in the Full Analysis Set CAT score at week 6 (mean change from baseline)	-1.5 units on a scale Interval -4.0 to 1.1
Mean Change From Baseline - CAT Score in the Full Analysis Set CAT score at week 12 (mean change from baseline)	-2.3 units on a scale Interval -4.0 to -0.6

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 1: (0) I never cough → (5) I cough all the time. CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (1, Cough) in the Full Analysis Set CAT question 1 (cough) at week 6 (mean change from baseline)	-0.2 units on a scale Interval -0.7 to 0.3
Mean Change From Baseline - CAT Individual Item (1, Cough) in the Full Analysis Set CAT question 1 (cough) at week 12 (mean change from baseline)	-0.3 units on a scale Interval -0.9 to 0.4

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 2: (0) I have no phlegm (mucus) in my chest at all → (5) My chest is completely full of phlegm (mucus) CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (2, Mucus) in the Full Analysis Set CAT question 2 (mucus) at week 6 (mean change from baseline)	0.0 units on a scale Interval -0.7 to 0.6
Mean Change From Baseline - CAT Individual Item (2, Mucus) in the Full Analysis Set CAT question 2 (mucus) at week 12 (mean change from baseline)	-0.5 units on a scale Interval -1.1 to 0.0

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 3: (0) My chest does not feel tight at all → (5) My chest feels very tight CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (3, Chest Tight) in the Full Analysis Set CAT question 3 (chest tight) at week 6 (mean change from baseline)	-0.3 units on a scale Interval -0.7 to 0.1
Mean Change From Baseline - CAT Individual Item (3, Chest Tight) in the Full Analysis Set CAT question 3 (chest tight) at week 12 (mean change from baseline)	-0.4 units on a scale Interval -0.8 to 0.1

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 4: (0) When I walk up a hill or one flight of stairs I am not breathless → (5) When I walk up a hill or one flight of stairs I am very breathless CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (4, Breathless) in the Full Analysis Set CAT question 4 (breathless) at week 6 (mean change from baseline)	-0.9 units on a scale Interval -1.6 to -0.2
Mean Change From Baseline - CAT Individual Item (4, Breathless) in the Full Analysis Set CAT question 4 (breathless) at week 12 (mean change from baseline)	-0.5 units on a scale Interval -1.0 to 0.0

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 5: (0) I am not limited doing any activities at home → (5) I am very limited doing activities at home CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (5, Activities) in the Full Analysis Set CAT question 5 (activities) at week 6 (mean change from baseline)	-0.2 units on a scale Interval -0.7 to 0.2
Mean Change From Baseline - CAT Individual Item (5, Activities) in the Full Analysis Set CAT question 5 (activities) at week 12 (mean change from baseline)	-0.3 units on a scale Interval -0.8 to 0.1

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 6: (0) I am confident leaving my home despite my lung condition → (5) I am not at all confident leaving my home because of my lung condition CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (6, Confidence) in the Full Analysis Set CAT question 6 (confidence) at week 6 (mean change from baseline)	0.1 units on a scale Interval -0.5 to 0.8
Mean Change From Baseline - CAT Individual Item (6, Confidence) in the Full Analysis Set CAT question 6 (confidence) at week 12 (mean change from baseline)	-0.2 units on a scale Interval -0.8 to 0.4

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 7: (0) I sleep soundly → (5) I don't sleep soundly because of my lung condition CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (7, Sleep) in the Full Analysis Set CAT question 7 (sleep) at week 6 (mean change from baseline)	0.0 units on a scale Interval -0.5 to 0.5
Mean Change From Baseline - CAT Individual Item (7, Sleep) in the Full Analysis Set CAT question 7 (sleep) at week 12 (mean change from baseline)	0.2 units on a scale Interval -0.2 to 0.5

SECONDARY outcome

Timeframe: At week 6 and week 12

Population: The full analysis set (FAS) included all subjects administered the study medication at visit 2 (day 1), and had a CAT completed at baseline and from at least one post-treatment visit.

The COPD Assessment Test (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.

Question 8: (0) I have lots of energy → (5) I have no energy at all CAT was self-completed at baseline, week 6 and week 12.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline - CAT Individual Item (8, Energy) in the Full Analysis Set CAT question 8 (energy) at week 6 (mean change from baseline)	0.0 units on a scale Interval -0.5 to 0.6
Mean Change From Baseline - CAT Individual Item (8, Energy) in the Full Analysis Set CAT question 8 (energy) at week 12 (mean change from baseline)	-0.2 units on a scale Interval -0.7 to 0.2

OTHER_PRE_SPECIFIED outcome

Timeframe: 12 Weeks

Population: The safety analysis set (SAS) included all subjects who were administered the study medication at day 1 (n=20). However, 2 subjects did not complete week 6 and week 12 visits and did not have a change from baseline calculated.

Vital signs were assessed at baseline, week 6 and week 12. Week 12 is reported. Determine any clinically significant change from baseline in systolic and diastolic blood pressure.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline in Blood Pressure in the Safety Analysis Set Systolic blood pressure at week 12 (mean change from baseline)	1.2 mmHg Standard Deviation 15.08
Mean Change From Baseline in Blood Pressure in the Safety Analysis Set Diastolic blood pressure at week 12 (mean change from baseline)	2.8 mmHg Standard Deviation 10.37

OTHER_PRE_SPECIFIED outcome

Timeframe: 12 Weeks

Population: The safety analysis set (SAS) included all subjects who were administered the study medication at day 1 (n=20). However, 2 subjects did not complete week 6 and week 12 visits and did not have a change from baseline calculated.

Vital signs were assessed at baseline, week 6 and week 12. Week 12 is reported. Determine any clinically significant change from baseline in pulse rate.

Outcome measures

Measure	Ensifentrine 3 mg BID n=18 Participants Inhaled ensifentrine twice daily (BID) for 12 weeks via standard jet nebulizer in addition to standard dual (LAMA/LABA) or triple (LAMA/LABA/ICS) therapy. Eligible subjects received ensifentrine.
Mean Change From Baseline in Pulse Rate in the Safety Analysis Set	2.9 bpm Standard Deviation 11.11

Adverse Events

Ensifentrine 3 mg BID

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Clinical Development

Verona Pharma

Phone: 844-341-9901

Email: clinicaltrials@veronapharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60