Trial Outcomes & Findings for Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients (NCT NCT02532764)
NCT ID: NCT02532764
Last Updated: 2019-02-06
Results Overview
Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
70 participants
Primary outcome timeframe
8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts
Results posted on
2019-02-06
Participant Flow
The enrollment of this clinical trial followed a staggered approach; a new SAD cohort was enrolled following review of the previous SAD cohort. The first MAD cohort opened following the review of the first two SAD cohorts. Subsequent MAD dosing cohorts were initiated following review of the corresponding supportive SAD and MAD cohorts.
Participant milestones
| Measure |
QR-010 SAD 6.25mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
QR-010 SAD 12.5 mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
QR-010 SAD 25 mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
QR-010 SAD 50 mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose Placebo: Normal Saline
|
QR-010 MAD 6.25
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Single Ascending Dose (SAD) Cohorts
STARTED
|
6
|
6
|
9
|
6
|
9
|
0
|
0
|
0
|
0
|
0
|
|
Single Ascending Dose (SAD) Cohorts
COMPLETED
|
6
|
6
|
9
|
6
|
9
|
0
|
0
|
0
|
0
|
0
|
|
Single Ascending Dose (SAD) Cohorts
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
|
Multiple Ascending Dose (MAD) Cohorst
STARTED
|
0
|
0
|
0
|
0
|
0
|
6
|
6
|
7
|
6
|
9
|
|
Multiple Ascending Dose (MAD) Cohorst
COMPLETED
|
0
|
0
|
0
|
0
|
0
|
6
|
6
|
6
|
5
|
8
|
|
Multiple Ascending Dose (MAD) Cohorst
NOT COMPLETED
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
1
|
1
|
1
|
Reasons for withdrawal
| Measure |
QR-010 SAD 6.25mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
QR-010 SAD 12.5 mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
QR-010 SAD 25 mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
QR-010 SAD 50 mg
QR-010 administered via inhalation as a single dose. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose Placebo: Normal Saline
|
QR-010 MAD 6.25
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks. QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalation.
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Multiple Ascending Dose (MAD) Cohorst
Met stopping criteria
|
0
|
0
|
0
|
0
|
0
|
0
|
0
|
1
|
1
|
1
|
Baseline Characteristics
Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients
Baseline characteristics by cohort
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 Participants
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
Total
n=70 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
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
6 Participants
n=10 Participants
|
6 Participants
n=115 Participants
|
7 Participants
n=6 Participants
|
6 Participants
n=6 Participants
|
9 Participants
n=64 Participants
|
70 Participants
n=17 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Age, Continuous
|
25.8 years
STANDARD_DEVIATION 8.01 • n=5 Participants
|
21.5 years
STANDARD_DEVIATION 2.95 • n=7 Participants
|
27.2 years
STANDARD_DEVIATION 7.31 • n=5 Participants
|
22.7 years
STANDARD_DEVIATION 3.88 • n=4 Participants
|
24.8 years
STANDARD_DEVIATION 8.26 • n=21 Participants
|
22.7 years
STANDARD_DEVIATION 2.66 • n=10 Participants
|
27.7 years
STANDARD_DEVIATION 10.42 • n=115 Participants
|
32.3 years
STANDARD_DEVIATION 10.63 • n=6 Participants
|
23.3 years
STANDARD_DEVIATION 4.18 • n=6 Participants
|
26.3 years
STANDARD_DEVIATION 6.69 • n=64 Participants
|
25.6 years
STANDARD_DEVIATION 7.35 • n=17 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
3 Participants
n=10 Participants
|
3 Participants
n=115 Participants
|
5 Participants
n=6 Participants
|
2 Participants
n=6 Participants
|
6 Participants
n=64 Participants
|
38 Participants
n=17 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
4 Participants
n=4 Participants
|
6 Participants
n=21 Participants
|
3 Participants
n=10 Participants
|
3 Participants
n=115 Participants
|
2 Participants
n=6 Participants
|
4 Participants
n=6 Participants
|
3 Participants
n=64 Participants
|
32 Participants
n=17 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
6 Participants
n=10 Participants
|
6 Participants
n=115 Participants
|
7 Participants
n=6 Participants
|
6 Participants
n=6 Participants
|
9 Participants
n=64 Participants
|
70 Participants
n=17 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
0 Participants
n=10 Participants
|
0 Participants
n=115 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=6 Participants
|
0 Participants
n=64 Participants
|
0 Participants
n=17 Participants
|
|
Region of Enrollment
Canada
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
2 participants
n=5 Participants
|
0 participants
n=4 Participants
|
0 participants
n=21 Participants
|
0 participants
n=10 Participants
|
0 participants
n=115 Participants
|
1 participants
n=6 Participants
|
0 participants
n=6 Participants
|
0 participants
n=64 Participants
|
3 participants
n=17 Participants
|
|
Region of Enrollment
Belgium
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
2 participants
n=21 Participants
|
1 participants
n=10 Participants
|
1 participants
n=115 Participants
|
0 participants
n=6 Participants
|
1 participants
n=6 Participants
|
2 participants
n=64 Participants
|
9 participants
n=17 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
4 participants
n=5 Participants
|
5 participants
n=4 Participants
|
1 participants
n=21 Participants
|
1 participants
n=10 Participants
|
1 participants
n=115 Participants
|
0 participants
n=6 Participants
|
1 participants
n=6 Participants
|
3 participants
n=64 Participants
|
18 participants
n=17 Participants
|
|
Region of Enrollment
Czechia
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
2 participants
n=21 Participants
|
0 participants
n=10 Participants
|
2 participants
n=115 Participants
|
0 participants
n=6 Participants
|
1 participants
n=6 Participants
|
0 participants
n=64 Participants
|
9 participants
n=17 Participants
|
|
Region of Enrollment
Denmark
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
0 participants
n=4 Participants
|
2 participants
n=21 Participants
|
1 participants
n=10 Participants
|
0 participants
n=115 Participants
|
2 participants
n=6 Participants
|
1 participants
n=6 Participants
|
1 participants
n=64 Participants
|
8 participants
n=17 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
2 participants
n=21 Participants
|
1 participants
n=10 Participants
|
1 participants
n=115 Participants
|
1 participants
n=6 Participants
|
0 participants
n=6 Participants
|
0 participants
n=64 Participants
|
7 participants
n=17 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
1 participants
n=4 Participants
|
0 participants
n=21 Participants
|
1 participants
n=10 Participants
|
0 participants
n=115 Participants
|
0 participants
n=6 Participants
|
1 participants
n=6 Participants
|
3 participants
n=64 Participants
|
8 participants
n=17 Participants
|
|
Region of Enrollment
France
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
1 participants
n=5 Participants
|
0 participants
n=4 Participants
|
0 participants
n=21 Participants
|
0 participants
n=10 Participants
|
0 participants
n=115 Participants
|
1 participants
n=6 Participants
|
0 participants
n=6 Participants
|
0 participants
n=64 Participants
|
2 participants
n=17 Participants
|
|
Region of Enrollment
Germany
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
0 participants
n=21 Participants
|
1 participants
n=10 Participants
|
1 participants
n=115 Participants
|
2 participants
n=6 Participants
|
1 participants
n=6 Participants
|
0 participants
n=64 Participants
|
6 participants
n=17 Participants
|
|
Weight (kg)
|
59.4 kg
STANDARD_DEVIATION 9.28 • n=5 Participants
|
64.8 kg
STANDARD_DEVIATION 2.26 • n=7 Participants
|
67.5 kg
STANDARD_DEVIATION 13.00 • n=5 Participants
|
57.7 kg
STANDARD_DEVIATION 9.70 • n=4 Participants
|
62.2 kg
STANDARD_DEVIATION 6.48 • n=21 Participants
|
66.8 kg
STANDARD_DEVIATION 6.87 • n=10 Participants
|
62.6 kg
STANDARD_DEVIATION 8.97 • n=115 Participants
|
67.1 kg
STANDARD_DEVIATION 4.35 • n=6 Participants
|
61.3 kg
STANDARD_DEVIATION 13.37 • n=6 Participants
|
59.6 kg
STANDARD_DEVIATION 9.19 • n=64 Participants
|
63.0 kg
STANDARD_DEVIATION 9.11 • n=17 Participants
|
|
BMI
|
22.1 kg/m2
STANDARD_DEVIATION 2.54 • n=5 Participants
|
24.1 kg/m2
STANDARD_DEVIATION 2.33 • n=7 Participants
|
23.0 kg/m2
STANDARD_DEVIATION 2.59 • n=5 Participants
|
21.5 kg/m2
STANDARD_DEVIATION 2.91 • n=4 Participants
|
21.4 kg/m2
STANDARD_DEVIATION 1.73 • n=21 Participants
|
23.5 kg/m2
STANDARD_DEVIATION 2.76 • n=10 Participants
|
22.8 kg/m2
STANDARD_DEVIATION 2.65 • n=115 Participants
|
23.6 kg/m2
STANDARD_DEVIATION 1.56 • n=6 Participants
|
21.3 kg/m2
STANDARD_DEVIATION 2.86 • n=6 Participants
|
20.9 kg/m2
STANDARD_DEVIATION 1.56 • n=64 Participants
|
22.3 kg/m2
STANDARD_DEVIATION 2.42 • n=17 Participants
|
|
FEV1
|
3.24 L
STANDARD_DEVIATION 0.877 • n=5 Participants
|
3.15 L
STANDARD_DEVIATION 0.666 • n=7 Participants
|
3.78 L
STANDARD_DEVIATION 1.092 • n=5 Participants
|
3.06 L
STANDARD_DEVIATION 0.700 • n=4 Participants
|
3.64 L
STANDARD_DEVIATION 0.748 • n=21 Participants
|
3.49 L
STANDARD_DEVIATION 0.590 • n=10 Participants
|
3.19 L
STANDARD_DEVIATION 0.600 • n=115 Participants
|
2.85 L
STANDARD_DEVIATION 0.577 • n=6 Participants
|
3.40 L
STANDARD_DEVIATION 0.993 • n=6 Participants
|
3.18 L
STANDARD_DEVIATION 0.778 • n=64 Participants
|
3.32 L
STANDARD_DEVIATION 0.792 • n=17 Participants
|
|
ppFEV1
|
92.3 percent
STANDARD_DEVIATION 13.31 • n=5 Participants
|
91.4 percent
STANDARD_DEVIATION 19.93 • n=7 Participants
|
97.3 percent
STANDARD_DEVIATION 20.39 • n=5 Participants
|
80.9 percent
STANDARD_DEVIATION 8.99 • n=4 Participants
|
91.8 percent
STANDARD_DEVIATION 12.91 • n=21 Participants
|
90.7 percent
STANDARD_DEVIATION 14.28 • n=10 Participants
|
89.0 percent
STANDARD_DEVIATION 14.21 • n=115 Participants
|
79.9 percent
STANDARD_DEVIATION 10.14 • n=6 Participants
|
84.7 percent
STANDARD_DEVIATION 13.59 • n=6 Participants
|
86.7 percent
STANDARD_DEVIATION 11.91 • n=64 Participants
|
88.8 percent
STANDARD_DEVIATION 14.54 • n=17 Participants
|
PRIMARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: All subjects who received either QR-010 or placebo
Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 Participants
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study
|
3 Participants
|
2 Participants
|
7 Participants
|
4 Participants
|
2 Participants
|
5 Participants
|
5 Participants
|
6 Participants
|
5 Participants
|
9 Participants
|
PRIMARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: All subjects who received either QR-010 or placebo.
Assessment of severity of treatment emergent adverse events (TEAEs). Severity is graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events Modified for CF (CTCAE v4.03). For events not present in this listing the following grading was applied: Mild: Asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Moderate: Minimal, local, or noninvasive intervention indicated; discomfort sufficient to reduce or interfere with daily activities; Severe: Medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization may be indicated; disabling; limits self-care with significant interference with daily activities; incapacitating with inability to perform self care activities of daily living; Life-threatening: Urgent intervention indicated; immediate risk of death.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 Participants
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Mild
|
3 Participants
|
2 Participants
|
5 Participants
|
4 Participants
|
2 Participants
|
5 Participants
|
4 Participants
|
5 Participants
|
1 Participants
|
5 Participants
|
|
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Moderate
|
0 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
4 Participants
|
3 Participants
|
|
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Severe
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Life-Threatening
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study
Death
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: All subjects who received either QR-010 or placebo. There were no Dose Limiting Toxicities (DLTs) reported in the SAD or MAD cohorts.
DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 Participants
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: All subjects who received either QR-010 or placebo.
Number of subjects experiencing at least one abnormality for the categories laboratory parameters, vital signs, ECG, spirometry and physical findings that were reported as treatment emergent adverse event with a relationship to study drug as either possibly, probably or definitely.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 Participants
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
Spirometry
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
2 Participants
|
1 Participants
|
|
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
physical findings
|
2 Participants
|
2 Participants
|
2 Participants
|
3 Participants
|
0 Participants
|
3 Participants
|
4 Participants
|
1 Participants
|
1 Participants
|
4 Participants
|
|
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
vital signs
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
ECG
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.
laboratory parameters
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: Study population included all subjects treated either with QR-010 or placebo. Quantifiable serum concentrations are only available for the 50mg SAD and 50 mg MAD cohorts.
Cmax: QR-010 maximum serum concentrations
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Maximum Serum Concentration
Dose 1
|
—
|
—
|
—
|
2.9 ng/mL
Standard Deviation 1.2
|
—
|
—
|
—
|
—
|
4.53 ng/mL
Standard Deviation 3.38
|
—
|
|
Maximum Serum Concentration
Week 4, Dose 12
|
—
|
—
|
—
|
NA ng/mL
Standard Deviation NA
for SAD cohorts, there is no Week 4, Dose 12 timepoint
|
—
|
—
|
—
|
—
|
3.63 ng/mL
Standard Deviation 2.73
|
—
|
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: Population included all subjects treated with either QR-010 or placebo. Quantifiable serum concentrations are only available for the 50mg SAD and MAD cohorts.
Tmax: Time to Cmax of QR-010 serum concentrations.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Time to Maximum Serum Concentration
Dose 1
|
—
|
—
|
—
|
0.6 hour
Interval 0.5 to 2.1
|
—
|
—
|
—
|
—
|
0.5 hour
Interval 0.5 to 3.0
|
—
|
|
Time to Maximum Serum Concentration
Week 4, Dose 12
|
—
|
—
|
—
|
NA hour
for SAD cohorts, there is no Week 4, Dose 12 timepoint
|
—
|
—
|
—
|
—
|
1.0 hour
Interval 1.0 to 2.0
|
—
|
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: The terminal half-life is not reported because the %AUC (Area Under the Curve) extrapolation was too large.
The terminal elimination half-life will be estimated by non-linear regression analysis of the terminal elimination slope
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: Quantifiable serum concentrations are only available for the 50mg SAD and MAD cohort. For the PK parameters of the SAD 6.25, 12.5 and 25mg cohorts, no quantifiable serum concentrations could be measured. For the 6.25, 12.5 and 25mg MAD cohorts, no PK profiling was performed due to the large number of samples below the limit of detection.
Area under the curve to the final sample with a concentration greater than lower limit of quantification (LLQ) will be calculated using the linear trapezoidal method
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 Participants
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Area Under the Curve to Final Sample [AUC(0-last)]
Dose 1
|
—
|
—
|
—
|
17.2 ng.hr/mL
Standard Deviation 8.2
|
—
|
—
|
—
|
—
|
19.0 ng.hr/mL
Standard Deviation 15.6
|
—
|
|
Area Under the Curve to Final Sample [AUC(0-last)]
Week 4, Dose 12
|
—
|
—
|
—
|
NA ng.hr/mL
Standard Deviation NA
for SAD cohorts, there is no Week 4, Dose 12 timepoint
|
—
|
—
|
—
|
—
|
17.0 ng.hr/mL
Standard Deviation 11.4
|
—
|
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: The Area Under the Curve to Infinity is not reported because the %AUC extrapolation was too large
AUC0-∞: Area under the curve to infinity will be calculated based on the last observed concentration Clast(obs) using formula: AUC0-∞=AUClast+Clast(obs)/λz
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsPopulation: The Serum Clearance is not reported because the %AUC extrapolation was too large.
CL: Serum clearance will be estimated using the formula: CL = Dose/AUC0-∞.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses. For 1 subject in the placebo group no data were available at Day 54. "Mean" in this table refers to "Adjusted Mean".
Patient Reported Outcome measure Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score (CFQ-R RSS). A higher score represents a better outcome. A minimal clinically important difference (MCID) in the respiratory domain (CFQ-R RSS) has been established in stable populations as 4.0 points, and a maximum score is 100 points. Mean values reported refer to ''adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=8 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in CFQ-R RSS
Day 15
|
-4.13 score on a scale
Standard Error 4.10
|
8.95 score on a scale
Standard Error 4.12
|
7.76 score on a scale
Standard Error 4.15
|
-0.80 score on a scale
Standard Error 4.51
|
-5.09 score on a scale
Standard Error 3.59
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS
Day 33
|
6.43 score on a scale
Standard Error 4.92
|
12.65 score on a scale
Standard Error 4.93
|
7.76 score on a scale
Standard Error 4.96
|
-3.02 score on a scale
Standard Error 5.40
|
-6.48 score on a scale
Standard Error 4.29
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS
Day 54
|
-10.24 score on a scale
Standard Error 6.56
|
0.61 score on a scale
Standard Error 6.57
|
5.91 score on a scale
Standard Error 6.59
|
-4.13 score on a scale
Standard Error 7.20
|
-10.31 score on a scale
Standard Error 5.87
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses.
Patient Reported Outcome measure Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score (CFQ-R RSS). A higher score represents a better outcome. A minimal clinically important difference (MCID) in the respiratory domain (CFQ-R RSS) has been established in stable populations as 4.0 points, and a maximum score is 100 points. Mean values reported refer to ''adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo
Day 15
|
0.96 score on a scale
Standard Error 5.46
|
14.03 score on a scale
Standard Error 5.49
|
12.85 score on a scale
Standard Error 5.55
|
4.29 score on a scale
Standard Error 5.73
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo
Day 33
|
12.91 score on a scale
Standard Error 6.53
|
19.13 score on a scale
Standard Error 6.56
|
14.24 score on a scale
Standard Error 6.60
|
3.46 score on a scale
Standard Error 6.87
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo
Day 54
|
0.07 score on a scale
Standard Error 8.81
|
10.92 score on a scale
Standard Error 8.83
|
16.22 score on a scale
Standard Error 8.87
|
6.18 score on a scale
Standard Error 9.26
|
—
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: Subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses, with ppFEV1 \<90% at baseline. For 1 subject in the placebo group no data were available at Day 54. "Mean" in this table refers to "Adjusted Mean".
Patient Reported Outcome measure Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score (CFQ-R RSS). A higher score represents a better outcome. A minimal clinically important difference (MCID) in the respiratory domain (CFQ-R RSS) has been established in stable populations as 4.0 points, and a maximum score is 100 points. Mean values reported refer to ''adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=3 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=4 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)
Day 54
|
-3.37 score on a scale
Standard Error 6.36
|
5.86 score on a scale
Standard Error 5.59
|
7.33 score on a scale
Standard Error 4.94
|
-3.65 score on a scale
Standard Error 5.52
|
-6.49 score on a scale
Standard Error 6.33
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)
Day 15
|
-3.37 score on a scale
Standard Error 5.51
|
8.64 score on a scale
Standard Error 4.87
|
9.56 score on a scale
Standard Error 4.28
|
-0.87 score on a scale
Standard Error 4.79
|
-4.77 score on a scale
Standard Error 4.82
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)
Day 33
|
11.45 score on a scale
Standard Error 7.36
|
15.58 score on a scale
Standard Error 6.44
|
8.44 score on a scale
Standard Error 5.71
|
-0.87 score on a scale
Standard Error 6.38
|
-11.72 score on a scale
Standard Error 6.41
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses, with ppFEV1 \<90% at Baseline.
Patient Reported Outcome measure Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score (CFQ-R RSS). A higher score represents a better outcome. A minimal clinically important difference (MCID) in the respiratory domain (CFQ-R RSS) has been established in stable populations as 4.0 points, and a maximum score is 100 points. Mean values reported refer to ''difference vs placebo in adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=3 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)
Day 15
|
1.41 score on a scale
Standard Error 7.29
|
13.41 score on a scale
Standard Error 6.95
|
14.33 score on a scale
Standard Error 6.49
|
3.90 score on a scale
Standard Error 6.74
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)
Day 33
|
23.17 score on a scale
Standard Error 9.73
|
27.30 score on a scale
Standard Error 9.17
|
20.16 score on a scale
Standard Error 8.62
|
10.84 score on a scale
Standard Error 9.01
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)
Day 54
|
3.12 score on a scale
Standard Error 8.93
|
12.35 score on a scale
Standard Error 8.58
|
13.82 score on a scale
Standard Error 8.08
|
2.84 score on a scale
Standard Error 8.33
|
—
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses.
Exploratory efficacy parameter, as measured by spirometry, and expressed in percent predicted FEV1 (ppFEV1). Mean values reported refer to ''adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=8 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in ppFEV1
(pre-dose) Day 15
|
0.06 percentage of predicted
Standard Error 1.96
|
2.50 percentage of predicted
Standard Error 1.96
|
-4.03 percentage of predicted
Standard Error 1.96
|
-1.85 percentage of predicted
Standard Error 2.14
|
-0.83 percentage of predicted
Standard Error 1.70
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1
(pre-dose) Day 26
|
0.46 percentage of predicted
Standard Error 2.64
|
3.22 percentage of predicted
Standard Error 2.64
|
-0.98 percentage of predicted
Standard Error 2.64
|
-1.39 percentage of predicted
Standard Error 2.89
|
-0.80 percentage of predicted
Standard Error 2.28
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1
Day 33
|
1.54 percentage of predicted
Standard Error 2.40
|
0.05 percentage of predicted
Standard Error 2.40
|
-2.17 percentage of predicted
Standard Error 2.40
|
-3.21 percentage of predicted
Standard Error 2.63
|
-2.70 percentage of predicted
Standard Error 2.08
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1
Day 54
|
1.68 percentage of predicted
Standard Error 2.88
|
-1.63 percentage of predicted
Standard Error 2.88
|
0.61 percentage of predicted
Standard Error 2.88
|
-2.43 percentage of predicted
Standard Error 3.15
|
-2.58 percentage of predicted
Standard Error 2.49
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses.
Exploratory efficacy parameter, as measured by spirometry, and expresssed in percent predicted FEV1 (ppFEV1). Mean values reported refer to''difference vs placebo in adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=6 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo
Day 15
|
0.89 percentage of predicted
Standard Error 2.59
|
3.32 percentage of predicted
Standard Error 2.59
|
-3.20 percentage of predicted
Standard Error 2.59
|
-1.02 percentage of predicted
Standard Error 2.73
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo
Day 26
|
1.26 percentage of predicted
Standard Error 3.49
|
4.02 percentage of predicted
Standard Error 3.49
|
-0.17 percentage of predicted
Standard Error 3.49
|
-0.59 percentage of predicted
Standard Error 3.68
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo
Day 33
|
4.24 percentage of predicted
Standard Error 3.18
|
2.75 percentage of predicted
Standard Error 3.18
|
0.53 percentage of predicted
Standard Error 3.18
|
-0.51 percentage of predicted
Standard Error 3.35
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo
Day 54
|
4.26 percentage of predicted
Standard Error 3.81
|
0.96 percentage of predicted
Standard Error 3.80
|
3.20 percentage of predicted
Standard Error 3.81
|
0.15 percentage of predicted
Standard Error 4.01
|
—
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses, with ppFEV1 \<90% at Baseline..
Exploratory efficacy parameter, as measured by spirometry, and expresssed in percent predicted FEV1. Mean values reported refer to ''adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=3 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=4 Participants
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)
Day 33
|
6.01 percentage of predicted
Standard Error 3.16
|
3.81 percentage of predicted
Standard Error 2.74
|
-0.68 percentage of predicted
Standard Error 2.44
|
-0.97 percentage of predicted
Standard Error 2.73
|
-4.18 percentage of predicted
Standard Error 2.76
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)
Day 15
|
-0.29 percentage of predicted
Standard Error 2.22
|
4.54 percentage of predicted
Standard Error 1.92
|
-3.45 percentage of predicted
Standard Error 1.71
|
-1.01 percentage of predicted
Standard Error 1.91
|
-3.40 percentage of predicted
Standard Error 1.95
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)
Day 26
|
4.21 percentage of predicted
Standard Error 4.05
|
7.15 percentage of predicted
Standard Error 3.51
|
0.89 percentage of predicted
Standard Error 3.13
|
-0.10 percentage of predicted
Standard Error 3.50
|
-3.76 percentage of predicted
Standard Error 3.52
|
—
|
—
|
—
|
—
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 15, Day 33, Day 54Population: Subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses, with ppFEV1 \<90% at Baseline.
Exploratory efficacy parameter, as measured by spirometry, and expresssed in percent predicted FEV1. Mean values reported refer to ''difference vs placebo in adjusted mean change from baseline'' values.
Outcome measures
| Measure |
QR-010 SAD 6.25 mg
n=3 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=5 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=4 Participants
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)
Day 15
|
3.11 percentage of predicted
Standard Error 2.99
|
7.93 percentage of predicted
Standard Error 2.76
|
-0.05 percentage of predicted
Standard Error 2.59
|
2.38 percentage of predicted
Standard Error 2.74
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)
Day 26
|
7.97 percentage of predicted
Standard Error 5.39
|
10.90 percentage of predicted
Standard Error 4.98
|
4.65 percentage of predicted
Standard Error 4.71
|
3.65 percentage of predicted
Standard Error 4.97
|
—
|
—
|
—
|
—
|
—
|
—
|
|
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)
Day 33
|
10.19 percentage of predicted
Standard Error 4.22
|
7.99 percentage of predicted
Standard Error 3.91
|
3.50 percentage of predicted
Standard Error 3.69
|
3.21 percentage of predicted
Standard Error 3.89
|
—
|
—
|
—
|
—
|
—
|
—
|
Adverse Events
QR-010 SAD 6.25 mg
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
QR-010 SAD 12.5 mg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
QR-010 SAD 25 mg
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
QR-010 SAD 50 mg
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Placebo SAD
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
QR-010 MAD 6.25 mg
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
QR-010 MAD 12.5 mg
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
QR-010 MAD 25 mg
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
QR-010 MAD 50 mg
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Placebo MAD
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
QR-010 SAD 6.25 mg
n=6 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 participants at risk
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 participants at risk
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • Number of events 1 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • Number of events 1 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Renal and urinary disorders
Calculus Urinary
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • Number of events 1 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
Other adverse events
| Measure |
QR-010 SAD 6.25 mg
n=6 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 12.5 mg
n=6 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 25 mg
n=9 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 SAD 50 mg
n=6 participants at risk
QR-010 administered via inhalation as a single dose
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo SAD
n=9 participants at risk
Placebo (normal saline) administered via inhalation as a single dose
Placebo: Normal Saline
|
QR-010 MAD 6.25 mg
n=6 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 12.5 mg
n=6 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 25 mg
n=7 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
QR-010 MAD 50 mg
n=6 participants at risk
QR-010 administered via inhalation three times weekly for four weeks.
QR-010: Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton
|
Placebo MAD
n=9 participants at risk
Placebo (normal saline) administered via inhalation three times weekly for four weeks.
Placebo: Normal Saline
|
|---|---|---|---|---|---|---|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
14.3%
1/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
General disorders
Pyrexia
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
28.6%
2/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
General disorders
Fatigue
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
14.3%
1/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Nervous system disorders
Headache
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
22.2%
2/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Gastrointestinal disorders
Dry Mouth
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
14.3%
1/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
3/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Infections and infestations
Oral candidiasis
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
14.3%
1/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Infections and infestations
Respiratory Tract Infection
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
14.3%
1/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Investigations
Forced expiratory volume decreased
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
22.2%
2/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
14.3%
1/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Psychiatric disorders
Agitation
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
50.0%
3/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
33.3%
2/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
22.2%
2/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
50.0%
3/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Skin and subcutaneous tissue disorders
Pruritus generalised
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
Injury, poisoning and procedural complications
Sunburn
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
General disorders
Chest pain
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
|
General disorders
Chest discomfort
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
16.7%
1/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/7 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
0.00%
0/6 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
11.1%
1/9 • For the SAD cohorts, from randomization up to Day 8 after dose administration. For the MAD cohorts, from randomization for at least 30 days after the last administration of the study treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Investigators are restricted from disclosure or results until after multicenter 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 45 days (varying per site). Sponsor may request redaction of confidential information.
- Publication restrictions are in place
Restriction type: OTHER