Trial Outcomes & Findings for Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation (NCT NCT02564354)
NCT ID: NCT02564354
Last Updated: 2020-09-24
Results Overview
The primary endpoint was the within-subject change from baseline in total chloride transport as measured by NPD, after the Chloride-free+isoproterenol solution (Cl-free+iso), and was based on the average measurements of both nostrils. To provide baseline stability, baseline was defined as the average of the two most recent pre-dose values, where each pre-dose value was the average of two nostrils. A negative change from baseline of Cl-free+iso shows an improvement.
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
18 participants
Primary outcome timeframe
Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment.
Results posted on
2020-09-24
Participant Flow
Subjects were screened and enrolled at 5 hospitals in the USA and Europe. Screenings for Cohort 1 and Cohort 2 occurred in parallel between between 19th October 2015 and 14th July 2016.
Participant milestones
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
Subjects homozygous for the F508del-CFTR (Cystic Fibrosis Transmembrane conductance Regulator) mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
8
|
|
Overall Study
COMPLETED
|
10
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation
Baseline characteristics by cohort
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
25.80 years
n=5 Participants
|
36.00 years
n=7 Participants
|
30.33 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
France
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
FEV1% predicted
|
74.22 Percent of the predicted value
n=5 Participants
|
73.55 Percent of the predicted value
n=7 Participants
|
73.92 Percent of the predicted value
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment.Population: Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement.
The primary endpoint was the within-subject change from baseline in total chloride transport as measured by NPD, after the Chloride-free+isoproterenol solution (Cl-free+iso), and was based on the average measurements of both nostrils. To provide baseline stability, baseline was defined as the average of the two most recent pre-dose values, where each pre-dose value was the average of two nostrils. A negative change from baseline of Cl-free+iso shows an improvement.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD).
Baseline
|
1.86 mV
Standard Deviation 3.360
|
-0.75 mV
Standard Deviation 3.868
|
|
Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD).
Week 2
|
-1.11 mV
Standard Deviation 8.062
|
1.66 mV
Standard Deviation 3.937
|
|
Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD).
Week 4
|
-1.92 mV
Standard Deviation 4.837
|
1.43 mV
Standard Deviation 2.644
|
|
Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD).
3 weeks post-treatment
|
-1.86 mV
Standard Deviation 6.736
|
2.27 mV
Standard Deviation 6.546
|
SECONDARY outcome
Timeframe: 2 and 4 weeks, and at 3 weeks post-treatment.Population: Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement.
This analysis is the proportion (amount) of subjects with an average Cl-free+iso actual value of -6.6 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The actual value of -6.6 mV is used to discriminate individuals with CF (\>-6.6 mV) from normal individuals (≤ -6.6 mV).
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subjects With a -6.6 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study.
Week 2
|
1 Participants
|
0 Participants
|
|
Number of Subjects With a -6.6 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study.
Week 4
|
1 Participants
|
0 Participants
|
|
Number of Subjects With a -6.6 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study.
3 weeks post-treatment
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 2 and 4 weeks, and at 3 weeks post-treatment.Population: Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement.
This analysis is the proportion of subjects with an average Cl-free+iso actual value of -4 mV or more negative after treatment (ie, responders), as measured by NPD and was based on the average measurements of both nostrils. The value of -4 mV is considered a clinically relevant response to treatment based on data from studies of other CFTR therapies.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study.
Week 4
|
2 Participants
|
1 Participants
|
|
Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study.
3 weeks post-treatment
|
4 Participants
|
0 Participants
|
|
Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study.
Week 2
|
3 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment.Population: Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement.
This endpoint was the within-subject change in Sodium transport (average Potential Difference prior to perfusion of any solution); this is the average of Potential Difference measured at five sites in the inferior meatus of the nose. A positive change from baseline shows an improvement.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study.
Baseline
|
-31.92 mV
Standard Deviation 8.777
|
-27.02 mV
Standard Deviation 11.412
|
|
Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study.
Week 2
|
-33.80 mV
Standard Deviation 9.922
|
-27.48 mV
Standard Deviation 9.230
|
|
Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study.
Week 4
|
-25.14 mV
Standard Deviation 10.809
|
-25.52 mV
Standard Deviation 10.445
|
|
Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study.
3 weeks post-treatment
|
-31.56 mV
Standard Deviation 7.791
|
-27.59 mV
Standard Deviation 6.291
|
SECONDARY outcome
Timeframe: 2 and 4 weeks, and at 3 weeks post-treatment.Population: Per Protocol Set: 3 subjects from Cohort 1 and 1 subject from Cohort 2 didn't meet the NPD entry criterion and were excluded from the per protocol analysis. Further 1 subject from Cohort 1 did not have an interpretable Week 4 NPD measurement.
The mean change in CFTR-mediated Total Chloride Transport compared to Baseline, per cohort; this is the mean of Potential Difference measured at five sites in the inferior meatus of the nose measured in millivolts (mV). A negative change from baseline shows an improvement.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=7 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
The Mean Change in CFTR-mediated Total Chloride Transport.
Week 2
|
-2.44 mV
Standard Error 2.00
|
1.88 mV
Standard Error 2.00
|
|
The Mean Change in CFTR-mediated Total Chloride Transport.
Week 4
|
-3.46 mV
Standard Error 1.88
|
1.65 mV
Standard Error 1.70
|
|
The Mean Change in CFTR-mediated Total Chloride Transport.
3 weeks post-treatment
|
-3.19 mV
Standard Error 2.26
|
2.49 mV
Standard Error 2.26
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study.
Number of subjects experiencing serious adverse events from baseline through End of Study.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subjects Experiencing Serious Adverse Events From Baseline Through End of Study.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study; incidence of discontinuations due to AEs from baseline through End of Study will be reported in the Adverse Events section. No discontinuations occurred.
Number of subject discontinuations due to AEs from baseline through End of Study. No discontinuations occurred.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subject Discontinuations Due to AEs From Baseline Through End of Study.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study.
Number of subjects experiencing at least one abnormality in laboratory parameters (chemistry, hematology and urinalysis) that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subjects With Abnormalities of Laboratory Parameters From Baseline Through End of Study.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study.
Number of subjects experiencing at least one abnormality vital signs \& oximetry that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subjects With Abnormalities of Vital Signs & Oximetry From Baseline Through End of Study.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study.
Number of subjects experiencing at least one abnormality in physical examination that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely, from baseline through End of Study.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Number of Subjects With Abnormalities of Physical Examinations From Baseline Through End of Study.
|
3 Participants
|
4 Participants
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study. 1 measurement was missing from 1 subject in Cohort 2 for Week 4.
The NERS was performed by site staff prior to each dose and as part of the NPD procedure; it is a set of scales ranging from 0 (no symptoms) to 3 (most severe symptoms) for assessing the severity of each of the following nasal symptoms, separately for each nostril: mucosal disruption, edema, erythema, polyp, secretions. The range of the total sum is 0-30.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Baseline
|
12.20 units on a scale
Standard Deviation 1.317
|
11.50 units on a scale
Standard Deviation 1.773
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 3
|
11.60 units on a scale
Standard Deviation 1.647
|
13.38 units on a scale
Standard Deviation 3.335
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 5
|
11.50 units on a scale
Standard Deviation 1.650
|
12.25 units on a scale
Standard Deviation 2.550
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Week 1
|
12.60 units on a scale
Standard Deviation 2.171
|
12.00 units on a scale
Standard Deviation 1.852
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 10
|
11.50 units on a scale
Standard Deviation 1.900
|
11.88 units on a scale
Standard Deviation 2.532
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 12
|
11.50 units on a scale
Standard Deviation 1.269
|
11.50 units on a scale
Standard Deviation 1.414
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Week 2
|
12.50 units on a scale
Standard Deviation 2.635
|
11.75 units on a scale
Standard Deviation 1.909
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 17
|
11.20 units on a scale
Standard Deviation 2.098
|
12.00 units on a scale
Standard Deviation 2.878
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 19
|
11.90 units on a scale
Standard Deviation 2.183
|
11.63 units on a scale
Standard Deviation 1.923
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Week 3
|
13.10 units on a scale
Standard Deviation 2.685
|
12.63 units on a scale
Standard Deviation 2.134
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Day 24
|
11.90 units on a scale
Standard Deviation 2.378
|
12.38 units on a scale
Standard Deviation 2.326
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
Week 4
|
13.00 units on a scale
Standard Deviation 2.828
|
11.71 units on a scale
Standard Deviation 1.890
|
|
Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study.
3 weeks post-treatment
|
12.30 units on a scale
Standard Deviation 2.541
|
12.75 units on a scale
Standard Deviation 3.327
|
SECONDARY outcome
Timeframe: 3 weeks post-treatment.Population: Safety population: all subjects enrolled in the study.
Subjects were asked to score a list of 22 symptoms on the SNOT-22 regarding social and emotional consequences. Outcomes were graded as 0 (no problem), to 5 (problem as bad as it could be). The list included: need to blow nose, sneezing, dripping nose, cough, postnasal drip, dense nasal drip, ear fullness, dizziness, ear pain, facial pain/pressure, difficulty falling asleep, waking at night, lack of a good night's sleep, waking up tired, fatigue, reduced productivity, reduced concentration, frustrated/restless/irritable, sad, embarrassed, decrease in smell and taste, and nasal obstruction. The range of the total sum is 0-110, with higher values indicating worse outcome.
Outcome measures
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 Participants
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 Participants
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study.
Baseline
|
14.90 units on a scale
Standard Deviation 5.896
|
19.13 units on a scale
Standard Deviation 17.707
|
|
Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study.
Week 2
|
13.60 units on a scale
Standard Deviation 8.369
|
15.38 units on a scale
Standard Deviation 18.601
|
|
Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study.
Week 4
|
14.50 units on a scale
Standard Deviation 11.048
|
14.13 units on a scale
Standard Deviation 16.084
|
|
Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study.
3 weeks post-treatment
|
10.70 units on a scale
Standard Deviation 8.301
|
14.50 units on a scale
Standard Deviation 10.915
|
Adverse Events
Cohort 1: Homozygous for the F508del-CFTR Mutation
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1: Homozygous for the F508del-CFTR Mutation
n=10 participants at risk
Subjects homozygous for the F508del-CFTR mutation were enrolled into Cohort 1 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
Cohort 2: Compound Heterozygous for the F508del-CFTR Mutation
n=8 participants at risk
Subjects compound heterozygous for the F508del-CFTR mutation were enrolled into Cohort 2 and received QR-010 10 mg (5 mg/250 μL saline per nostril) via bilateral intranasal administration three times a week for 4 weeks for a total of 12 doses.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Gastrointestinal disorders
Constipation
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Gastrointestinal disorders
Gastritis
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Gastrointestinal disorders
Glossitis
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Gastrointestinal disorders
Nausea
|
20.0%
2/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
General disorders
Fatigue
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
50.0%
4/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
General disorders
Malaise
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
General disorders
Pyrexia
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
37.5%
3/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Hepatobiliary disorders
Galbladder pain
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Infections and infestations
Nasopharyngitis
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Infections and infestations
Rhinitis
|
30.0%
3/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Infections and infestations
Staphylococcal infection
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Infections and infestations
Upper respiratory tract infection
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Injury, poisoning and procedural complications
Wound
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Nervous system disorders
Headache
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
25.0%
2/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Nervous system disorders
Migraine
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
2/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
37.5%
3/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Increased viscosity of bronchial secretion
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Intranasal hypoaesthesia
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal mucosal erosion
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal sinus discomfort
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Paranasal sinus hypersecretion
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
20.0%
2/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
30.0%
3/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
25.0%
2/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
10.0%
1/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
0.00%
0/8 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/10 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
12.5%
1/8 • Number of events 1 • After the subject had provided informed consent, but prior to initiation of IMP, only SAEs caused by a protocol-mandated intervention were collected. After initiation of IMP, all AEs and SAEs, regardless of attribution, were collected until at least 30 days following the last administration of IMP or initiation of new therapy, whichever was earlier.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Investigators are restricted from disclosure or results until after multi-center publication or 12 months after the completion of the Study at all participating research centers. Sponsor can review results communication prior to public release and can embargo communications regarding trial results for a period that is more than 60 days (varying per site). Sponsor may request redaction of confidential information.
- Publication restrictions are in place
Restriction type: OTHER