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A Study of RPL554 in Patients With Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02919995
Recruitment Status : Completed
First Posted : September 30, 2016
Results First Posted : March 20, 2019
Last Update Posted : May 14, 2019
Sponsor:
Collaborator:
Cystic Fibrosis Trust
Information provided by (Responsible Party):
Verona Pharma plc

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: RPL554
Drug: Placebo
Enrollment 10
Recruitment Details  
Pre-assignment Details 16 patients were screened; six patients failed screening. Patients had to discontinue long acting bronchodilators on the day prior to screening and short acting bronchodilators for 8 hours before all spirometry assessments
Arm/Group Title Higher Dose RPL554/Lower Dose RPL554/Placebo Lower Dose RPL554/Placebo/Higher Dose RPL554 Higher Dose RPL554/Plaebo/Lower Dose RPL554 Lower Dose RPL554/Higher Dose RPL554/Placebo Placebo/Higher Dose RPL554/Lower Dose RPL554 Placebo/Lower Dose RPL554/Higher Dose RPL554
Hide Arm/Group Description

Single dose of inhaled 6 mg RPL554 in Period 1, Single inhaled dose of 1.5 mg RPL554 in Period 2, placebo in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554 in Period 1, single inhaled dose of placebo in Period 2, single inhaled dose of 6 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single inhaled dose of 6 mg RPL554 in Period 1, single inhaled dose of placebo in Period 2, single inhaled dose of 1.5 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single inhaled dose of 1.5 mg RPL554 in Period 1, single inhaled dose of 6 mg RPL554 in Period 2, single inhaled dose of placebo in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single inhaled dose of placebo in Period 1, single inhaled dose of 6 mg RPL554 in Period 2, single inhaled dose of 1.5 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single inhaled dose of placebo in Period 1, single inhaled dose of 1.5 mg RPL554 in Period 2, single inhaled dose of 6 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Period Title: Overall Study
Started 1 2 2 2 1 2
Completed 1 2 2 2 1 1
Not Completed 0 0 0 0 0 1
Reason Not Completed
Adverse Event             0             0             0             0             0             1
Arm/Group Title Higher Dose RPL554/Lower Dose RPL554/Placebo Lower Dose RPL554/Placebo/Higher Dose RPL554 Higher Dose RPL554/Placebo/Lower Dose RPL554 Lower Dose RPL554/Higher Dose RPL554/Placebo Placebo/Higher Dose RPL554/Lower Dose RPL554 Placebo/Lower Dose RPL554/Higher Dose RPL554 Total
Hide Arm/Group Description

Single dose of inhaled 6 mg RPL554 in Period 1, single dose of inhaled 1.5 mg RPL554 in Period 2, single inhaled dose of placebo in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554 in Period 1, single dose of inhaled placebo RPL554 in Period 2, single dose of 6 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single dose of inhaled 6 mg RPL554 in Period 1, single dose of inhaled placebo in Period 2, single dose of inhaled 1.5 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554 in Period 1, single dose of inhaled 6 mg RPL554 in Period 2, single inhaled dose of placebo in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single dose of inhaled placebo in Period 1, single dose of inhaled 6 mg RPL554 in Period 2, single dose of inhaled 1.5 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Single dose of inhaled placebo in Period 1, single dose of inhaled 1.5 mg RPL554 in Period 2, single dose of inhaled 6 mg RPL554 in Period 3

RPL554: RPL554 suspension administered using a nebuliser Placebo: Placebo solution administered using a nebuliser

Total of all reporting groups
Overall Number of Baseline Participants 1 2 2 2 1 2 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 2 participants 2 participants 2 participants 1 participants 2 participants 10 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
1
 100.0%
2
 100.0%
2
 100.0%
2
 100.0%
1
 100.0%
2
 100.0%
10
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 2 participants 2 participants 2 participants 1 participants 2 participants 10 participants
Female
0
   0.0%
0
   0.0%
2
 100.0%
1
  50.0%
0
   0.0%
1
  50.0%
4
  40.0%
Male
1
 100.0%
2
 100.0%
0
   0.0%
1
  50.0%
1
 100.0%
1
  50.0%
6
  60.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 2 participants 2 participants 2 participants 1 participants 2 participants 10 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
1
 100.0%
2
 100.0%
2
 100.0%
2
 100.0%
1
 100.0%
2
 100.0%
10
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title AUC by Dose
Hide Description Area under the curve (AUC)
Time Frame Pre dose, 15 and 30 minutes and 1, 2, 4, 6, 8 and 24 hours post dose after each treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All randomised patients with blood sampling performed after at least one dose of RPL554 and with data sufficient to calculate pharmacokinetic parameters.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: pg*h/mL
7699  (2965.7) 2342  (1029.9)
2.Primary Outcome
Title Maximum Plasma Concentration After Each Dose
Hide Description Maximum plasma concentration (Cmax) after a single dose of RPL554
Time Frame Pre dose, 15 and 30 minutes and 1, 2, 4, 6, 8 and 24 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
All randomised patients with blood sampling performed after at least one dose of RPL554 and with data sufficient to calculate pharmacokinetic parameters.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: pg/mL
828.3  (256.1) 270.1  (91.9)
3.Primary Outcome
Title Time to Maximum Plasma Concentration After Each Dose
Hide Description Time to maximum concentration (Tmax) after a single dose of RPL554
Time Frame Pre dose, 15 and 30 minutes and 1, 2, 4, 6, 8 and 24 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
All randomised patients with blood sampling performed after at least one dose of RPL554 and with data sufficient to calculate pharmacokinetic parameters.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: hours
1.53  (0.66) 1.33  (0.62)
4.Primary Outcome
Title Half Life for Each Dose
Hide Description Half life (t1/2) of RPL554
Time Frame Pre dose, 15 and 30 minutes and 1, 2, 4, 6, 8 and 24 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
All randomised patients with blood sampling performed after at least one dose of RPL554 and with data sufficient to calculate pharmacokinetic parameters.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Overall Number of Participants Analyzed 9 9
Mean (Standard Deviation)
Unit of Measure: Hours
10.14  (3.1) 7.52  (3.3)
5.Secondary Outcome
Title Peak FEV1 for Each Treatment
Hide Description Maximum Forced expired volume in one second (FEV1) measured using spirometry
Time Frame Pre dose and 15 and 30 minutes and 1, 2 and 4 hours post dose after treatment
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized patients with sufficient data collected after intake of study treatment to compute the pharmacodynamic parameters on at least two study visits.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554 Placebo
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Inhaled placebo dose

Placebo: Placebo solution administered using a nebuliser

Overall Number of Participants Analyzed 9 10 10
Mean (Standard Deviation)
Unit of Measure: Liters
2.384  (0.73) 2.247  (0.72) 2.256  (0.71)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Lower Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0196
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.038
Confidence Interval (2-Sided) 95%
1.007 to 1.07
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0802
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.024
Confidence Interval (2-Sided) 95%
0.997 to 1.052
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Lower Dose RPL554
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3487
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 0.986
Confidence Interval (2-Sided) 95%
0.957 to 1.017
Estimation Comments [Not Specified]
6.Secondary Outcome
Title AUC FEV1(0-4h)
Hide Description Area under the curve for FEV1 over 4 hours measured using spirometry
Time Frame Pre dose and 15 and 30 minutes and 1, 2 and 4 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized patients with sufficient data collected after intake of study treatment to compute the pharmacodynamic parameters on at least two study visits.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554 Placebo
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Inhaled placebo dose

Placebo: Placebo solution administered using a nebuliser

Overall Number of Participants Analyzed 9 10 10
Mean (Standard Deviation)
Unit of Measure: Liters
2.313  (0.73) 2.194  (0.72) 2.133  (0.73)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Lower Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0043
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.072
Confidence Interval (2-Sided) 95%
1.026 to 1.12
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0109
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.055
Confidence Interval (2-Sided) 95%
1.014 to 1.096
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Lower Dose RPL554
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4306
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 0.984
Confidence Interval (2-Sided) 95%
0.942 to 1.027
Estimation Comments [Not Specified]
7.Secondary Outcome
Title AUC FEV1(0-6h)
Hide Description Area under the curve FEV1 over 6 hours measured using spirometry
Time Frame Pre dose and 15 and 30 minutes and 1, 2, 4 and 6 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
all randomized patients with sufficient data collected after intake of study treatment to compute the pharmacodynamic parameters on at least two study visits.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554 Placebo
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Inhaled placebo dose

Placebo: Placebo solution administered using a nebuliser

Overall Number of Participants Analyzed 9 10 10
Mean (Standard Deviation)
Unit of Measure: Liters
2.304  (0.74) 2.188  (0.73) 2.133  (0.73)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Lower Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0064
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.065
Confidence Interval (2-Sided) 95%
1.021 to 1.11
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0149
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.049
Confidence Interval (2-Sided) 95%
1.011 to 1.089
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Lower Dose RPL554
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.466
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 0.945
Confidence Interval (2-Sided) 95%
0.945 to 1.027
Estimation Comments [Not Specified]
8.Secondary Outcome
Title AUC FEV1(0-8h)
Hide Description Area under the curve for FEV1 over 8 hours measured using spirometry
Time Frame pre dose and 15 and 30 minutes and 1, 2, 4, 6 and 8 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
All randomised patients with sufficient data collected after intake of study treatment to compute the pharmacodynamic parameters on at least two study visits.
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554 Placebo
Hide Arm/Group Description:

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Inhaled placebo dose

Placebo: Placebo solution administered using a nebuliser

Overall Number of Participants Analyzed 9 10 10
Mean (Standard Deviation)
Unit of Measure: Liters
2.287  (0.75) 2.185  (0.74) 2.130  (0.74)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Lower Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0093
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.061
Confidence Interval (2-Sided) 95%
1.017 to 1.107
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0333
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 1.042
Confidence Interval (2-Sided) 95%
1.004 to 1.082
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Higher Dose RPL554, Lower Dose RPL554
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3693
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Contrast ratio
Estimated Value 0.982
Confidence Interval (2-Sided) 95%
0.941 to 1.024
Estimation Comments [Not Specified]
9.Secondary Outcome
Title FVC
Hide Description Forced vital capacity (FVC) measured using spirometry
Time Frame Over 24 hours after treatment
Outcome Measure Data Not Reported
10.Secondary Outcome
Title Breath Samples
Hide Description Exhaled breath pH
Time Frame 8 and 24 hours after treatment
Outcome Measure Data Not Reported
11.Secondary Outcome
Title Laboratory Safety Tests 1
Hide Description Biochemistry panel parameters
Time Frame Screening and end of study
Outcome Measure Data Not Reported
12.Secondary Outcome
Title Laboratory Safety Tests 2
Hide Description Haematology panel parameters
Time Frame Screening and end of study
Outcome Measure Data Not Reported
13.Secondary Outcome
Title Laboratory Safety Tests 3
Hide Description Urinalysis measured by urine dipstick
Time Frame Screening and end of study
Outcome Measure Data Not Reported
14.Secondary Outcome
Title Vital Signs 1
Hide Description Pulse rate after 5 minutes supine
Time Frame Over 8 hours after treatment
Outcome Measure Data Not Reported
15.Secondary Outcome
Title Vital Signs 2
Hide Description Blood pressure after 5 minutes supine
Time Frame Over 8 hours after treatment
Outcome Measure Data Not Reported
16.Secondary Outcome
Title ECG 1
Hide Description Heart rate
Time Frame Over 8 hours after treatment
Outcome Measure Data Not Reported
17.Secondary Outcome
Title ECG 2
Hide Description QT interval
Time Frame Over 8 hours after treatment
Outcome Measure Data Not Reported
18.Other Pre-specified Outcome
Title Sputum Rheology
Hide Description Rheological analysis for interleukin 8, tumour necrosis factor alpha and myeloperoxidase
Time Frame 8 and 12 hours after treatment
Outcome Measure Data Not Reported
19.Other Pre-specified Outcome
Title Sputum Measurements
Hide Description Levels of inflammatory mediators
Time Frame 8 and 12 hours after treatment
Outcome Measure Data Not Reported
Time Frame From informed consent through study completion, up to 52 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Higher Dose RPL554 Lower Dose RPL554 Placebo
Hide Arm/Group Description

Single dose of inhaled 6 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Single dose of inhaled 1.5 mg RPL554

RPL554: RPL554 suspension administered using a nebuliser

Inhaled placebo dose

Placebo: Placebo solution administered using a nebuliser

All-Cause Mortality
Higher Dose RPL554 Lower Dose RPL554 Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/9 (0.00%)      0/10 (0.00%)      0/10 (0.00%)    
Hide Serious Adverse Events
Higher Dose RPL554 Lower Dose RPL554 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/9 (0.00%)      1/10 (10.00%)      0/10 (0.00%)    
Infections and infestations       
exacerbation of cystic fibrosis * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
1
Term from vocabulary, MedDRA 19.1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Higher Dose RPL554 Lower Dose RPL554 Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   6/9 (66.67%)      6/10 (60.00%)      3/10 (30.00%)    
Cardiac disorders       
Tachycardia * 1  2/9 (22.22%)  2 2/10 (20.00%)  2 1/10 (10.00%)  1
Gastrointestinal disorders       
Abdominal pain lower * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
Dry mouth * 1  0/9 (0.00%)  0 0/10 (0.00%)  0 1/10 (10.00%)  1
Nausea * 1  1/9 (11.11%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
General disorders       
Chest discomfort * 1  1/9 (11.11%)  1 1/10 (10.00%)  1 0/10 (0.00%)  0
Immune system disorders       
Drug hypersensitivity * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
Infections and infestations       
Infective exacerbation of cystic fibrosis * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
Oral candidiasis * 1  1/9 (11.11%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
Investigations       
Forced expiratory volume decreased * 1  1/9 (11.11%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
Pulmonary function test decreased * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 1/10 (10.00%)  1
Musculoskeletal and connective tissue disorders       
Back pain * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
Nervous system disorders       
Headache * 1  0/9 (0.00%)  0 1/10 (10.00%)  1 0/10 (0.00%)  0
Syncope * 1  0/9 (0.00%)  0 0/10 (0.00%)  0 1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders       
Cough * 1  2/9 (22.22%)  2 1/10 (10.00%)  2 0/10 (0.00%)  0
Nasal congestion * 1  0/9 (0.00%)  0 0/10 (0.00%)  0 1/10 (10.00%)  1
Rhinorrhoea * 1  1/9 (11.11%)  1 0/10 (0.00%)  0 0/10 (0.00%)  0
1
Term from vocabulary, MedDRA 19.1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Investigator shall not be permitted to present at symposia, national or regional professional meetings, nor publish in journals, theses or dissertations, or otherwise of their own choosing, methods and results of the Clinical Trial without the prior written approval of the Sponsor. Any such approved presentation or publication shall acknowledge the Sponsor as the sponsor of the Clinical Trial and owner of the results of the Clinical Trial.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Brian Maurer
Organization: Verona Pharma plc
Phone: +19147675037
EMail: brian.maurer@veronapharma.com
Layout table for additonal information
Responsible Party: Verona Pharma plc
ClinicalTrials.gov Identifier: NCT02919995    
Other Study ID Numbers: RPL554-010-2015
2015-004263-36 ( EudraCT Number )
First Submitted: September 21, 2016
First Posted: September 30, 2016
Results First Submitted: December 7, 2018
Results First Posted: March 20, 2019
Last Update Posted: May 14, 2019