Trial Outcomes & Findings for A Phase 3 Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD (NCT NCT04542057)

NCT ID: NCT04542057

Last Updated: 2023-10-16

Results Overview

Forced spirometry maneuvers including the FEV1 were used to assess pulmonary function. Average FEV1 AUC0-12h was defined as AUC over 12 hours of the FEV1, divided by 12 hours. Baseline FEV1 is the mean of the 2 measurements taken before study medication on the day of first dosing, that is, \<=40 minutes pre-dose on Day 1. Spirometry assessments were performed in accordance with American Thoracic Society (ATS)/European Respiratory Society (ERS) guidelines.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

790 participants

Primary outcome timeframe

Baseline (40 minutes before first administration on Day 1) and Week 12

Results posted on

2023-10-16

Participant Flow

This Phase 3, randomized, double-blind, placebo-controlled study was conducted in patients with moderate to severe chronic obstructive pulmonary disease (COPD) at 130 study centers in Belgium, Bulgaria, Canada, Denmark, Estonia, Hungary, Poland, Slovakia, Spain, United States of America between 22 September 2020 and 06 July 2022. Patients were randomized in a 5:3 ratio, stratified by smoking status and background medication use, manner to receive either ensifentrine or placebo.

Patients were screened for eligibility before entering a 28-day run in period to ensure a stable COPD treatment regimen and to collect baseline information on symptoms and rescue medication use.

Participant milestones

Participant milestones
Measure	Ensifentrine 3 mg twice daily via standard jet nebulizer	Placebo twice daily via standard jet nebulizer
Overall Study STARTED	499	291
Overall Study Received Treatment	498	291
Overall Study COMPLETED	393	218
Overall Study NOT COMPLETED	106	73

Reasons for withdrawal

Reasons for withdrawal
Measure	Ensifentrine 3 mg twice daily via standard jet nebulizer	Placebo twice daily via standard jet nebulizer
Overall Study Death	3	1
Overall Study COPD exacerbation withdrawal criteria	5	6
Overall Study Coronavirus disease 2019 (COVID-19)	10	7
Overall Study Adverse Event	15	6
Overall Study COVID-19 adverse event	6	4
Overall Study Lack of Efficacy	2	5
Overall Study Study terminated by sponsor	1	0
Overall Study Investigator discretion	2	1
Overall Study Withdrawal by Subject	52	30
Overall Study Lost to Follow-up	8	11
Overall Study Other	2	2

Baseline Characteristics

A Phase 3 Trial to Evaluate the Safety and Efficacy of Ensifentrine in Patients With COPD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ensifentrine n=499 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer	Total n=790 Participants Total of all reporting groups
Age, Continuous	65.0 years STANDARD_DEVIATION 7.38 • n=5 Participants	65.3 years STANDARD_DEVIATION 7.30 • n=7 Participants	65.1 years STANDARD_DEVIATION 7.35 • n=5 Participants
Sex: Female, Male Female	254 Participants n=5 Participants	153 Participants n=7 Participants	407 Participants n=5 Participants
Sex: Female, Male Male	245 Participants n=5 Participants	138 Participants n=7 Participants	383 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaska native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	24 Participants n=5 Participants	11 Participants n=7 Participants	35 Participants n=5 Participants
Race/Ethnicity, Customized White	472 Participants n=5 Participants	276 Participants n=7 Participants	748 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	26 Participants n=5 Participants	14 Participants n=7 Participants	40 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	473 Participants n=5 Participants	277 Participants n=7 Participants	750 Participants n=5 Participants
Region of Enrollment Belgium	4 participants n=5 Participants	1 participants n=7 Participants	5 participants n=5 Participants
Region of Enrollment Bulgaria	78 participants n=5 Participants	53 participants n=7 Participants	131 participants n=5 Participants
Region of Enrollment Canada	7 participants n=5 Participants	6 participants n=7 Participants	13 participants n=5 Participants
Region of Enrollment Denmark	3 participants n=5 Participants	1 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Estonia	18 participants n=5 Participants	2 participants n=7 Participants	20 participants n=5 Participants
Region of Enrollment Hungary	32 participants n=5 Participants	20 participants n=7 Participants	52 participants n=5 Participants
Region of Enrollment Poland	38 participants n=5 Participants	20 participants n=7 Participants	58 participants n=5 Participants
Region of Enrollment Slovakia	13 participants n=5 Participants	5 participants n=7 Participants	18 participants n=5 Participants
Region of Enrollment Spain	25 participants n=5 Participants	9 participants n=7 Participants	34 participants n=5 Participants
Region of Enrollment United States	281 participants n=5 Participants	174 participants n=7 Participants	455 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (40 minutes before first administration on Day 1) and Week 12

Population: The modified Intent-to-Treat (mITT) population set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to randomized treatment.

Outcome measures

Outcome measures
Measure	Ensifentrine n=498 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
Least Square (LS) Mean Change From Baseline in Average Forced Expiratory Volume in 1 Second (FEV1) Area Under the Curve Over 12 Hours (AUC0-12h) at Week 12	0.0480 liters Standard Error 0.00941	-0.0462 liters Standard Error 0.01236

SECONDARY outcome

Timeframe: Baseline (40 minutes before first administration on Day 1), post-dose on Day 1, Weeks 6, 12, and 24

Population: The mITT population set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to randomized treatment.

Forced spirometry maneuvers including the FEV1 were used to assess pulmonary function. Peak FEV1 is the maximum value in the 4 hours after dosing. Baseline FEV1 is the mean of the 2 measurements taken before study medication on the day of first dosing, that is, \<=40 minutes pre-dose on Day 1. Spirometry assessments were performed in accordance with ATS/ERS guidelines.

Outcome measures

Outcome measures
Measure	Ensifentrine n=498 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline FEV1 to Peak FEV1 at Day 1 and Weeks 6, 12 and 24 Day 1 (Post-dose)	0.2369 liters Standard Error 0.00601	0.0801 liters Standard Error 0.00784
LS Mean Change From Baseline FEV1 to Peak FEV1 at Day 1 and Weeks 6, 12 and 24 Week 6	0.2158 liters Standard Error 0.00969	0.0636 liters Standard Error 0.01276
LS Mean Change From Baseline FEV1 to Peak FEV1 at Day 1 and Weeks 6, 12 and 24 Week 12	0.1945 liters Standard Error 0.01012	0.0482 liters Standard Error 0.01349
LS Mean Change From Baseline FEV1 to Peak FEV1 at Day 1 and Weeks 6, 12 and 24 Week 24	0.1957 liters Standard Error 0.01099	0.0434 liters Standard Error 0.01475

SECONDARY outcome

Timeframe: Baseline (average of 7 days before first administration on Day 1) and Weeks 6, 12, and 24

The E-RS scale consists of 11 questions, with 3 sub-domains of: breathlessness, cough and sputum, and chest symptoms. The E-RS sub-domain score was calculated as the sum from the relevant questions. The E-RS total score was derived as the sum of the raw scores of the 11 items ranging from 0 to 40. Higher scores indicates severe respiratory symptoms. Scores were derived weekly as the mean over 7 days prior to the visit, using only days where data was recorded. The E-RS was collected daily by electronic diary (e-diary). Baseline is the mean over the 7 days prior to the first intake of study medication, using only days where data was recorded.

Outcome measures

Outcome measures
Measure	Ensifentrine n=492 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline to the Mean Weekly Evaluating-Respiratory Symptoms (E-RS) Total Score at Weeks 6, 12 and 24 Week 6	-1.938 units on a scale Standard Error 0.2086	-0.614 units on a scale Standard Error 0.2763
LS Mean Change From Baseline to the Mean Weekly Evaluating-Respiratory Symptoms (E-RS) Total Score at Weeks 6, 12 and 24 Week 12	-2.051 units on a scale Standard Error 0.2245	-1.161 units on a scale Standard Error 0.2963
LS Mean Change From Baseline to the Mean Weekly Evaluating-Respiratory Symptoms (E-RS) Total Score at Weeks 6, 12 and 24 Week 24	-2.146 units on a scale Standard Error 0.2557	-1.529 units on a scale Standard Error 0.3365

SECONDARY outcome

Timeframe: Baseline (40 minutes before first administration on Day 1) and Weeks 6, 12, and 24

The SGRQ questionnaire consists of 17 questions, split into 2 parts. Part 1 consisted of the first 8 questions and was related to the symptoms subdomain. The remaining 9 questions were in Part 2, which were related to the activity and impacts subdomains. The total score was calculated by dividing the summed weights by the maximum possible weight for all items in the questionnaire and expressing the result as a percentage. Score ranging from 0 to 100 and higher scores indicated a worse outcome. Baseline is the score calculated on Day 1 prior to 4 hour post-dose spirometry.

Outcome measures

Outcome measures
Measure	Ensifentrine n=489 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=286 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline in the St. George's Respiratory Questionnaire (SGRQ) Total Score at Weeks 6, 12 and 24 Week 6	-3.602 units on a scale Standard Error 0.5902	-1.890 units on a scale Standard Error 0.7692
LS Mean Change From Baseline in the St. George's Respiratory Questionnaire (SGRQ) Total Score at Weeks 6, 12 and 24 Week 12	-4.019 units on a scale Standard Error 0.6171	-2.942 units on a scale Standard Error 0.8168
LS Mean Change From Baseline in the St. George's Respiratory Questionnaire (SGRQ) Total Score at Weeks 6, 12 and 24 Week 24	-4.532 units on a scale Standard Error 0.6840	-4.054 units on a scale Standard Error 0.9084

SECONDARY outcome

Timeframe: Baseline (40 minutes before first administration on Day 1) and Weeks 6, 12, and 24

Forced spirometry maneuvers including the FEV1 were used to assess pulmonary function. Morning trough FEV1 was the last value collected prior to the morning dose. Baseline FEV1 is the mean of the two measurements taken before study medication on the day of first dosing, that is, \<=40 minutes pre-dose on day 1. Spirometry assessments were performed in accordance with ATS/ERS guidelines.

Outcome measures

Outcome measures
Measure	Ensifentrine n=498 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline FEV1 to Morning Trough FEV1 at Weeks 6, 12 and 24 Week 6	0.0177 liters Standard Error 0.00899	-0.0261 liters Standard Error 0.01181
LS Mean Change From Baseline FEV1 to Morning Trough FEV1 at Weeks 6, 12 and 24 Week 12	0.0057 liters Standard Error 0.00957	-0.0435 liters Standard Error 0.01266
LS Mean Change From Baseline FEV1 to Morning Trough FEV1 at Weeks 6, 12 and 24 Week 24	-0.0066 liters Standard Error 0.01006	-0.0318 liters Standard Error 0.01323

SECONDARY outcome

Timeframe: Baseline (40 minutes before first administration on Day 1), post-dose on Day 1, Weeks 6, 12, and 24

Forced spirometry maneuvers including the FEV1 were used to assess pulmonary function. Average FEV1 AUC0-4h was defined as area under the curve over 4 hours of the FEV1, divided by 4 hours. Baseline FEV1 is the mean of the 2 measurements taken before study medication on the day of first dosing, that is, \<=40 minutes pre-dose on Day 1. Spirometry assessments were performed in accordance with ATS/ERS guidelines.

Outcome measures

Outcome measures
Measure	Ensifentrine n=498 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline in Average FEV1 Area Under the Curve Over 4 Hours (AUC0-4h) at Day 1 and Weeks 6, 12 and 24 Day 1 (Post-dose)	0.1556 liters Standard Error 0.00527	0.0063 liters Standard Error 0.00686
LS Mean Change From Baseline in Average FEV1 Area Under the Curve Over 4 Hours (AUC0-4h) at Day 1 and Weeks 6, 12 and 24 Week 6	0.1357 liters Standard Error 0.00932	-0.0082 liters Standard Error 0.01209
LS Mean Change From Baseline in Average FEV1 Area Under the Curve Over 4 Hours (AUC0-4h) at Day 1 and Weeks 6, 12 and 24 Week 12	0.1148 liters Standard Error 0.00943	-0.0209 liters Standard Error 0.01260
LS Mean Change From Baseline in Average FEV1 Area Under the Curve Over 4 Hours (AUC0-4h) at Day 1 and Weeks 6, 12 and 24 Week 24	0.1148 liters Standard Error 0.01036	-0.0248 liters Standard Error 0.01381

SECONDARY outcome

Timeframe: Weeks 6, 12 and 24

The SGRQ questionnaire consists of 17 questions, split into 2 parts. Part 1 consisted of the first 8 questions and was related to the symptoms subdomain. The remaining 9 questions were in Part 2, which were related to the activity and impacts subdomains. The total score was calculated by dividing the summed weights by the maximum possible weight for all items in the questionnaire and expressing the result as a percentage. Responder was a patient with an improvement from baseline in SGRQ total score of 4 or more. Percentage of SGRQ responders are reported.

Outcome measures

Outcome measures
Measure	Ensifentrine n=489 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=286 Participants twice daily via standard jet nebulizer
Percentage of SGRQ Responders at Weeks 6, 12 and 24 Week 6	44.0 percentage of patients	39.5 percentage of patients
Percentage of SGRQ Responders at Weeks 6, 12 and 24 Week 12	45.2 percentage of patients	43.0 percentage of patients
Percentage of SGRQ Responders at Weeks 6, 12 and 24 Week 24	45.4 percentage of patients	50.3 percentage of patients

SECONDARY outcome

Timeframe: Baseline (average of 7 days before first administration on Day 1) and Weeks 6, 12, and 24

Use of rescue medication (albuterol/salbutamol) per week was calculated as the LS mean use daily over 7 days. Daily rescue medication use was collected in an e-diary throughout the study. Baseline is the mean over the 7 days prior to the first intake of study medication, calculated as the sum of puffs taken, divided by number of days data has been recorded.

Outcome measures

Outcome measures
Measure	Ensifentrine n=492 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline to the Mean Weekly Rescue Medication Use at Weeks 6, 12 and 24 Week 6	-0.530 avg number of rescue medication puffs Standard Error 0.0883	-0.191 avg number of rescue medication puffs Standard Error 0.1165
LS Mean Change From Baseline to the Mean Weekly Rescue Medication Use at Weeks 6, 12 and 24 Week 12	-0.573 avg number of rescue medication puffs Standard Error 0.0745	-0.288 avg number of rescue medication puffs Standard Error 0.0976
LS Mean Change From Baseline to the Mean Weekly Rescue Medication Use at Weeks 6, 12 and 24 Week 24	-0.485 avg number of rescue medication puffs Standard Error 0.0871	-0.346 avg number of rescue medication puffs Standard Error 0.1143

SECONDARY outcome

Timeframe: Weeks 6, 12 and 24

The TDI is a questionnaire that focused on 3 sub-domains: functional impairment, magnitude of task and magnitude of effort. Sub-domain score was calculated as the sum from the related questions. Total score was calculated as the sum of the sub-domain scores. The TDI measures the change in dyspnea severity from the baseline as measured by the baseline dyspnea index. It was rated by 7 grades ranging from -3 (major deterioration) to +3 (major improvement). Higher scores indicate better outcome. Change from baseline was assessed with the Baseline Dyspnea Index.

Outcome measures

Outcome measures
Measure	Ensifentrine n=484 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=286 Participants twice daily via standard jet nebulizer
LS Mean Transition Dyspnea Index (TDI) Questionnaire Total Score at Weeks 6, 12 and 24 Week 6	1.6 units on a scale Standard Error 0.12	0.9 units on a scale Standard Error 0.16
LS Mean Transition Dyspnea Index (TDI) Questionnaire Total Score at Weeks 6, 12 and 24 Week 12	1.8 units on a scale Standard Error 0.13	1.2 units on a scale Standard Error 0.18
LS Mean Transition Dyspnea Index (TDI) Questionnaire Total Score at Weeks 6, 12 and 24 Week 24	2.2 units on a scale Standard Error 0.15	1.3 units on a scale Standard Error 0.20

SECONDARY outcome

Timeframe: Baseline (40 minutes before first administration on Day 1) and Week 12

Forced spirometry maneuvers including the FEV1 were used to assess pulmonary function. Evening trough FEV1 was the value collected at 12 hours post-morning dose and prior to the evening dose. Baseline FEV1 is the mean of the 2 measurements taken before study medication on the day of first dosing, that is, \<=40 minutes pre-dose on day 1. Spirometry assessments were performed in accordance with ATS/ERS guidelines.

Outcome measures

Outcome measures
Measure	Ensifentrine n=498 Participants 3 mg twice daily via standard jet nebulizer	Placebo n=291 Participants twice daily via standard jet nebulizer
LS Mean Change From Baseline FEV1 to Evening Trough FEV1 at Week 12	-0.0246 liters Standard Error 0.01079	-0.0783 liters Standard Error 0.01358

Adverse Events

Ensifentrine

Serious events: 28 serious events

Other events: 68 other events

Deaths: 4 deaths

Placebo

Serious events: 17 serious events

Other events: 46 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Ensifentrine n=498 participants at risk 3 mg twice daily via standard jet nebulizer	Placebo n=291 participants at risk twice daily via standard jet nebulizer
Infections and infestations COVID-19	3.2% 16/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	3.4% 10/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Infections and infestations Urinary tract infection	1.4% 7/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.7% 5/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Infections and infestations Nasopharyngitis	1.8% 9/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.0% 3/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Infections and infestations Sinusitis	1.0% 5/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	0.00% 0/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Respiratory, thoracic and mediastinal disorders Dyspnea	0.60% 3/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.4% 4/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Gastrointestinal disorders Diarrhea	1.6% 8/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	0.69% 2/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Musculoskeletal and connective tissue disorders Back pain	1.6% 8/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.7% 5/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Nervous system disorders Headache	2.0% 10/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	2.4% 7/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Cardiac disorders Angina pectoris	0.20% 1/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.4% 4/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Vascular disorders Hypertension	1.0% 5/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	0.34% 1/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Ear and labyrinth disorders Vertigo	0.20% 1/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.0% 3/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Infections and infestations Tooth Abcess	0.00% 0/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.4% 4/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.
Gastrointestinal disorders Gastrooesophageal reflux disease	0.40% 2/498 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.	1.0% 3/291 • Treatment-emergent adverse events (TEAEs) were collected from the first dose of study treatment up to 10 days after the final study visit at Week 24, approximately 25 weeks. The safety analysis set included all patients in the randomized set who received at least 1 dose (or partial dose) of study medication, and patients were classified according to treatment received. The TEAEs defined as adverse events that started or worsened in severity on or after the first dose of study treatment are reported.

Additional Information

Chief Medical Officer

Verona Pharma plc

Phone: See email

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place