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Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis

Status
Completed
Phases
Phase 1Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT02532764
Enrollment
70
Registered
2015-08-26
Start date
2015-06-30
Completion date
2017-09-14
Last updated
2019-02-06

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis, ΔF508, RNA therapies, antisense oligonucleotide, CFTR, F508del, CF, RNA therapy

Brief summary

A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

Detailed description

The purpose of this study is to evaluate the safety, tolerability, and to determine the pharmacokinetics of QR-010 administered via inhalation in adult homozygous for ΔF508 Cystic Fibrosis.

Interventions

DRUGQR-010

Single-stranded RNA antisense oligonucleotide in aqueous solution for oral inhalaton

DRUGPlacebo

Normal Saline

Sponsors

European Commission
CollaboratorOTHER
ProQR Therapeutics
Lead SponsorINDUSTRY

Study design

Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No

Inclusion criteria

* Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of \> 60 mmol/L * Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation * Body mass index (BMI) ≥ 17 kg/m2 * Non-smoking for a minimum of two years * FEV1 ≥70% of predicted normal for age, gender, and height, at Screening * Stable lung function * Adequate hepatic and renal function

Exclusion criteria

* Breast-feeding or pregnant * Use of lumacaftor or ivacaftor * Use of any investigational drug or device * History of lung transplantation * Hemoptysis

Design outcomes

Primary

MeasureTime frameDescription
Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsNumber of subjects experiencing at least one treatment emergent adverse events (TEAEs)
Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsDLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.
Severity of Treatment Emergent Adverse Events From Baseline Through End of Study8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsAssessment 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.

Secondary

MeasureTime frameDescription
Maximum Serum Concentration8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsCmax: QR-010 maximum serum concentrations
Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsNumber 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.
Time to Maximum Serum Concentration8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsTmax: Time to Cmax of QR-010 serum concentrations.
Terminal Half-life (T1/2)8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsThe terminal elimination half-life will be estimated by non-linear regression analysis of the terminal elimination slope
Area Under the Curve to Final Sample [AUC(0-last)]8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsArea 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
Area Under the Curve to Infinity [AUC(0-∞)]8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsAUC0-∞: Area under the curve to infinity will be calculated based on the last observed concentration Clast(obs) using formula: AUC0-∞=AUClast+Clast(obs)/λz
Serum Clearance (CL)8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohortsCL: Serum clearance will be estimated using the formula: CL = Dose/AUC0-∞.

Other

MeasureTime frameDescription
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 15, Day 33, Day 54Patient 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.
Adjusted Mean Change From Baseline in CFQ-R RSSDay 15, Day 33, Day 54Patient 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.
Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 15, Day 33, Day 54Patient 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.
Adjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 15, Day 33, Day 54Exploratory 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.
Adjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 15, Day 33, Day 54Exploratory efficacy parameter, as measured by spirometry, and expresssed in percent predicted FEV1. Mean values reported refer to ''adjusted mean change from baseline'' values.
Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 15, Day 33, Day 54Exploratory 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.
Adjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 15, Day 33, Day 54Patient 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.
Adjusted Mean Change From Baseline in ppFEV1Day 15, Day 33, Day 54Exploratory efficacy parameter, as measured by spirometry, and expressed in percent predicted FEV1 (ppFEV1). Mean values reported refer to ''adjusted mean change from baseline'' values.

Countries

Belgium, Canada, Czechia, Denmark, France, Germany, Italy, Spain, United Kingdom, United States

Participant flow

Recruitment details

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.

Participants by arm

ArmCount
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
6
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
6
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
9
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 inhalaton
6
Placebo SAD
Placebo (normal saline) administered via inhalation as a single dose Placebo: Normal Saline
9
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
6
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
6
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
7
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
6
Placebo MAD
Placebo (normal saline) administered via inhalation three times weekly for four weeks. Placebo: Normal Saline
9
Total70

Withdrawals & dropouts

PeriodReasonFG000FG001FG002FG003FG004FG005FG006FG007FG008FG009
Multiple Ascending Dose (MAD) CohorstMet stopping criteria0000000111

Baseline characteristics

CharacteristicPlacebo MADQR-010 MAD 50 mgQR-010 MAD 25 mgQR-010 MAD 12.5 mgQR-010 MAD 6.25 mgPlacebo SADQR-010 SAD 50 mgQR-010 SAD 25 mgQR-010 SAD 12.5 mgQR-010 SAD 6.25 mgTotal
Age, Categorical
<=18 years
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Age, Categorical
>=65 years
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Age, Categorical
Between 18 and 65 years
9 Participants6 Participants7 Participants6 Participants6 Participants9 Participants6 Participants9 Participants6 Participants6 Participants70 Participants
Age, Continuous26.3 years
STANDARD_DEVIATION 6.69
23.3 years
STANDARD_DEVIATION 4.18
32.3 years
STANDARD_DEVIATION 10.63
27.7 years
STANDARD_DEVIATION 10.42
22.7 years
STANDARD_DEVIATION 2.66
24.8 years
STANDARD_DEVIATION 8.26
22.7 years
STANDARD_DEVIATION 3.88
27.2 years
STANDARD_DEVIATION 7.31
21.5 years
STANDARD_DEVIATION 2.95
25.8 years
STANDARD_DEVIATION 8.01
25.6 years
STANDARD_DEVIATION 7.35
BMI20.9 kg/m2
STANDARD_DEVIATION 1.56
21.3 kg/m2
STANDARD_DEVIATION 2.86
23.6 kg/m2
STANDARD_DEVIATION 1.56
22.8 kg/m2
STANDARD_DEVIATION 2.65
23.5 kg/m2
STANDARD_DEVIATION 2.76
21.4 kg/m2
STANDARD_DEVIATION 1.73
21.5 kg/m2
STANDARD_DEVIATION 2.91
23.0 kg/m2
STANDARD_DEVIATION 2.59
24.1 kg/m2
STANDARD_DEVIATION 2.33
22.1 kg/m2
STANDARD_DEVIATION 2.54
22.3 kg/m2
STANDARD_DEVIATION 2.42
FEV13.18 L
STANDARD_DEVIATION 0.778
3.40 L
STANDARD_DEVIATION 0.993
2.85 L
STANDARD_DEVIATION 0.577
3.19 L
STANDARD_DEVIATION 0.6
3.49 L
STANDARD_DEVIATION 0.59
3.64 L
STANDARD_DEVIATION 0.748
3.06 L
STANDARD_DEVIATION 0.7
3.78 L
STANDARD_DEVIATION 1.092
3.15 L
STANDARD_DEVIATION 0.666
3.24 L
STANDARD_DEVIATION 0.877
3.32 L
STANDARD_DEVIATION 0.792
ppFEV186.7 percent
STANDARD_DEVIATION 11.91
84.7 percent
STANDARD_DEVIATION 13.59
79.9 percent
STANDARD_DEVIATION 10.14
89.0 percent
STANDARD_DEVIATION 14.21
90.7 percent
STANDARD_DEVIATION 14.28
91.8 percent
STANDARD_DEVIATION 12.91
80.9 percent
STANDARD_DEVIATION 8.99
97.3 percent
STANDARD_DEVIATION 20.39
91.4 percent
STANDARD_DEVIATION 19.93
92.3 percent
STANDARD_DEVIATION 13.31
88.8 percent
STANDARD_DEVIATION 14.54
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Asian
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Black or African American
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants0 Participants
Race (NIH/OMB)
White
9 Participants6 Participants7 Participants6 Participants6 Participants9 Participants6 Participants9 Participants6 Participants6 Participants70 Participants
Region of Enrollment
Belgium
2 participants1 participants0 participants1 participants1 participants2 participants0 participants0 participants1 participants1 participants9 participants
Region of Enrollment
Canada
0 participants0 participants1 participants0 participants0 participants0 participants0 participants2 participants0 participants0 participants3 participants
Region of Enrollment
Czechia
0 participants1 participants0 participants2 participants0 participants2 participants0 participants0 participants2 participants2 participants9 participants
Region of Enrollment
Denmark
1 participants1 participants2 participants0 participants1 participants2 participants0 participants1 participants0 participants0 participants8 participants
Region of Enrollment
France
0 participants0 participants1 participants0 participants0 participants0 participants0 participants1 participants0 participants0 participants2 participants
Region of Enrollment
Germany
0 participants1 participants2 participants1 participants1 participants0 participants0 participants0 participants1 participants0 participants6 participants
Region of Enrollment
Italy
0 participants0 participants1 participants1 participants1 participants2 participants0 participants0 participants1 participants1 participants7 participants
Region of Enrollment
United Kingdom
3 participants1 participants0 participants0 participants1 participants0 participants1 participants1 participants0 participants1 participants8 participants
Region of Enrollment
United States
3 participants1 participants0 participants1 participants1 participants1 participants5 participants4 participants1 participants1 participants18 participants
Sex: Female, Male
Female
6 Participants2 Participants5 Participants3 Participants3 Participants3 Participants2 Participants5 Participants5 Participants4 Participants38 Participants
Sex: Female, Male
Male
3 Participants4 Participants2 Participants3 Participants3 Participants6 Participants4 Participants4 Participants1 Participants2 Participants32 Participants
Weight (kg)59.6 kg
STANDARD_DEVIATION 9.19
61.3 kg
STANDARD_DEVIATION 13.37
67.1 kg
STANDARD_DEVIATION 4.35
62.6 kg
STANDARD_DEVIATION 8.97
66.8 kg
STANDARD_DEVIATION 6.87
62.2 kg
STANDARD_DEVIATION 6.48
57.7 kg
STANDARD_DEVIATION 9.7
67.5 kg
STANDARD_DEVIATION 13
64.8 kg
STANDARD_DEVIATION 2.26
59.4 kg
STANDARD_DEVIATION 9.28
63.0 kg
STANDARD_DEVIATION 9.11

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
EG002
affected / at risk
EG003
affected / at risk
EG004
affected / at risk
EG005
affected / at risk
EG006
affected / at risk
EG007
affected / at risk
EG008
affected / at risk
EG009
affected / at risk
deaths
Total, all-cause mortality
0 / 60 / 60 / 90 / 60 / 90 / 60 / 60 / 70 / 60 / 9
other
Total, other adverse events
3 / 62 / 67 / 94 / 62 / 95 / 65 / 66 / 75 / 69 / 9
serious
Total, serious adverse events
0 / 60 / 60 / 90 / 60 / 90 / 60 / 60 / 71 / 61 / 9

Outcome results

Primary

Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.

DLT's were defined as an allergic reaction, acute bronchospasm or acute AEs of interest requiring (immediate) medical intervention.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: All subjects who received either QR-010 or placebo. There were no Dose Limiting Toxicities (DLTs) reported in the SAD or MAD cohorts.

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
QR-010 SAD 6.25 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 SAD 12.5 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 SAD 25 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 SAD 50 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
Placebo SADIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 MAD 6.25 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 MAD 12.5 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 MAD 25 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
QR-010 MAD 50 mgIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
Placebo MADIncidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit.0 Participants
Primary

Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study

Number of subjects experiencing at least one treatment emergent adverse events (TEAEs)

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: All subjects who received either QR-010 or placebo

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
QR-010 SAD 6.25 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study3 Participants
QR-010 SAD 12.5 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study2 Participants
QR-010 SAD 25 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study7 Participants
QR-010 SAD 50 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study4 Participants
Placebo SADIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study2 Participants
QR-010 MAD 6.25 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study5 Participants
QR-010 MAD 12.5 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study5 Participants
QR-010 MAD 25 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study6 Participants
QR-010 MAD 50 mgIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study5 Participants
Placebo MADIncidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study9 Participants
Primary

Severity of Treatment Emergent Adverse Events From Baseline Through End of Study

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.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: All subjects who received either QR-010 or placebo.

ArmMeasureGroupValue (COUNT_OF_PARTICIPANTS)
QR-010 SAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 SAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild3 Participants
QR-010 SAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 SAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 SAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate0 Participants
QR-010 SAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate0 Participants
QR-010 SAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 SAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 SAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild2 Participants
QR-010 SAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 SAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 SAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 SAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate2 Participants
QR-010 SAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild5 Participants
QR-010 SAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 SAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 SAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild4 Participants
QR-010 SAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 SAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate0 Participants
QR-010 SAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
Placebo SADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
Placebo SADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate0 Participants
Placebo SADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
Placebo SADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild2 Participants
Placebo SADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 MAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild5 Participants
QR-010 MAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate0 Participants
QR-010 MAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 MAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 MAD 6.25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 MAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild4 Participants
QR-010 MAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 MAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate1 Participants
QR-010 MAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 MAD 12.5 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 MAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 MAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate0 Participants
QR-010 MAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 MAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere1 Participants
QR-010 MAD 25 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild5 Participants
QR-010 MAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere0 Participants
QR-010 MAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
QR-010 MAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
QR-010 MAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate4 Participants
QR-010 MAD 50 mgSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild1 Participants
Placebo MADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyLife-Threatening0 Participants
Placebo MADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyModerate3 Participants
Placebo MADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudySevere1 Participants
Placebo MADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyMild5 Participants
Placebo MADSeverity of Treatment Emergent Adverse Events From Baseline Through End of StudyDeath0 Participants
Secondary

Area Under the Curve to Final Sample [AUC(0-last)]

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

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: 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.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 50 mgArea Under the Curve to Final Sample [AUC(0-last)]Dose 117.2 ng.hr/mLStandard Deviation 8.2
QR-010 SAD 50 mgArea Under the Curve to Final Sample [AUC(0-last)]Week 4, Dose 12NA ng.hr/mL—
QR-010 MAD 50 mgArea Under the Curve to Final Sample [AUC(0-last)]Dose 119.0 ng.hr/mLStandard Deviation 15.6
QR-010 MAD 50 mgArea Under the Curve to Final Sample [AUC(0-last)]Week 4, Dose 1217.0 ng.hr/mLStandard Deviation 11.4
Secondary

Area Under the Curve to Infinity [AUC(0-∞)]

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

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: The Area Under the Curve to Infinity is not reported because the %AUC extrapolation was too large

Secondary

Maximum Serum Concentration

Cmax: QR-010 maximum serum concentrations

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: 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.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 50 mgMaximum Serum ConcentrationDose 12.9 ng/mLStandard Deviation 1.2
QR-010 SAD 50 mgMaximum Serum ConcentrationWeek 4, Dose 12NA ng/mL—
QR-010 MAD 50 mgMaximum Serum ConcentrationDose 14.53 ng/mLStandard Deviation 3.38
QR-010 MAD 50 mgMaximum Serum ConcentrationWeek 4, Dose 123.63 ng/mLStandard Deviation 2.73
Secondary

Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.

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.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: All subjects who received either QR-010 or placebo.

ArmMeasureGroupValue (COUNT_OF_PARTICIPANTS)
QR-010 SAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 SAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
QR-010 SAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 SAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings2 Participants
QR-010 SAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 SAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 SAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 SAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 SAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
QR-010 SAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings2 Participants
QR-010 SAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 SAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
QR-010 SAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings2 Participants
QR-010 SAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 SAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 SAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings3 Participants
QR-010 SAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 SAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 SAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 SAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
Placebo SADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
Placebo SADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
Placebo SADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
Placebo SADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
Placebo SADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings0 Participants
QR-010 MAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 MAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 MAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 MAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings3 Participants
QR-010 MAD 6.25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
QR-010 MAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings4 Participants
QR-010 MAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 MAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 MAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 MAD 12.5 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
QR-010 MAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 MAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters1 Participants
QR-010 MAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry0 Participants
QR-010 MAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 MAD 25 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings1 Participants
QR-010 MAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters0 Participants
QR-010 MAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
QR-010 MAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry2 Participants
QR-010 MAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
QR-010 MAD 50 mgNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings1 Participants
Placebo MADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.ECG0 Participants
Placebo MADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.Spirometry1 Participants
Placebo MADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.vital signs0 Participants
Placebo MADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.laboratory parameters3 Participants
Placebo MADNumber of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings.physical findings4 Participants
Secondary

Serum Clearance (CL)

CL: Serum clearance will be estimated using the formula: CL = Dose/AUC0-∞.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: The Serum Clearance is not reported because the %AUC extrapolation was too large.

Secondary

Terminal Half-life (T1/2)

The terminal elimination half-life will be estimated by non-linear regression analysis of the terminal elimination slope

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: The terminal half-life is not reported because the %AUC (Area Under the Curve) extrapolation was too large.

Secondary

Time to Maximum Serum Concentration

Tmax: Time to Cmax of QR-010 serum concentrations.

Time frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts

Population: Population included all subjects treated with either QR-010 or placebo. Quantifiable serum concentrations are only available for the 50mg SAD and MAD cohorts.

ArmMeasureGroupValue (MEDIAN)
QR-010 SAD 50 mgTime to Maximum Serum ConcentrationDose 10.6 hour
QR-010 SAD 50 mgTime to Maximum Serum ConcentrationWeek 4, Dose 12NA hour
QR-010 MAD 50 mgTime to Maximum Serum ConcentrationDose 10.5 hour
QR-010 MAD 50 mgTime to Maximum Serum ConcentrationWeek 4, Dose 121.0 hour
Other Pre-specified

Adjusted Mean Change From Baseline in CFQ-R RSS

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.

Time frame: Day 15, Day 33, Day 54

Population: 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.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 54-10.24 score on a scaleStandard Error 6.56
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 336.43 score on a scaleStandard Error 4.92
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 15-4.13 score on a scaleStandard Error 4.1
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 540.61 score on a scaleStandard Error 6.57
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 158.95 score on a scaleStandard Error 4.12
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 3312.65 score on a scaleStandard Error 4.93
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 337.76 score on a scaleStandard Error 4.96
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 157.76 score on a scaleStandard Error 4.15
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 545.91 score on a scaleStandard Error 6.59
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 15-0.80 score on a scaleStandard Error 4.51
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 54-4.13 score on a scaleStandard Error 7.2
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSSDay 33-3.02 score on a scaleStandard Error 5.4
Placebo SADAdjusted Mean Change From Baseline in CFQ-R RSSDay 54-10.31 score on a scaleStandard Error 5.87
Placebo SADAdjusted Mean Change From Baseline in CFQ-R RSSDay 33-6.48 score on a scaleStandard Error 4.29
Placebo SADAdjusted Mean Change From Baseline in CFQ-R RSSDay 15-5.09 score on a scaleStandard Error 3.59
Other Pre-specified

Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo

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.

Time frame: Day 15, Day 33, Day 54

Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 150.96 score on a scaleStandard Error 5.46
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 540.07 score on a scaleStandard Error 8.81
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 3312.91 score on a scaleStandard Error 6.53
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 1514.03 score on a scaleStandard Error 5.49
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 5410.92 score on a scaleStandard Error 8.83
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 3319.13 score on a scaleStandard Error 6.56
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 3314.24 score on a scaleStandard Error 6.6
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 1512.85 score on a scaleStandard Error 5.55
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 5416.22 score on a scaleStandard Error 8.87
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 154.29 score on a scaleStandard Error 5.73
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 546.18 score on a scaleStandard Error 9.26
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to PlaceboDay 333.46 score on a scaleStandard Error 6.87
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.059295% CI: [-0.54, 26.35]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.007495% CI: [5.62, 32.64]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.040895% CI: [0.64, 27.83]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.619395% CI: [-10.69, 17.6]Mixed Models Analysis
Other Pre-specified

Adjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup 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.

Time frame: Day 15, Day 33, Day 54

Population: 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.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 151.41 score on a scaleStandard Error 7.29
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 543.12 score on a scaleStandard Error 8.93
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 3323.17 score on a scaleStandard Error 9.73
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 1513.41 score on a scaleStandard Error 6.95
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 5412.35 score on a scaleStandard Error 8.58
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 3327.30 score on a scaleStandard Error 9.17
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 3320.16 score on a scaleStandard Error 8.62
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 1514.33 score on a scaleStandard Error 6.49
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 5413.82 score on a scaleStandard Error 8.08
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 153.90 score on a scaleStandard Error 6.74
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 542.84 score on a scaleStandard Error 8.33
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 3310.84 score on a scaleStandard Error 9.01
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.032195% CI: [2.29, 44.04]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.0195% CI: [7.64, 46.96]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.034695% CI: [1.68, 38.64]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline CFQ-R RSS as outcome variable and including treatment, baseline CFQ-R RSS value, time and interaction between time and treatment as covariates.p-value: 0.248595% CI: [-8.47, 30.16]Mixed Models Analysis
Other Pre-specified

Adjusted Mean Change From Baseline in CFQ-R RSS (Subgroup 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 ''adjusted mean change from baseline'' values.

Time frame: Day 15, Day 33, Day 54

Population: 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.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 15-3.37 score on a scaleStandard Error 5.51
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 54-3.37 score on a scaleStandard Error 6.36
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 3311.45 score on a scaleStandard Error 7.36
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 3315.58 score on a scaleStandard Error 6.44
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 158.64 score on a scaleStandard Error 4.87
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 545.86 score on a scaleStandard Error 5.59
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 338.44 score on a scaleStandard Error 5.71
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 159.56 score on a scaleStandard Error 4.28
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 547.33 score on a scaleStandard Error 4.94
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 15-0.87 score on a scaleStandard Error 4.79
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 54-3.65 score on a scaleStandard Error 5.52
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 33-0.87 score on a scaleStandard Error 6.38
Placebo SADAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 33-11.72 score on a scaleStandard Error 6.41
Placebo SADAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 15-4.77 score on a scaleStandard Error 4.82
Placebo SADAdjusted Mean Change From Baseline in CFQ-R RSS (Subgroup ppFEV1 <90% at Baseline)Day 54-6.49 score on a scaleStandard Error 6.33
Other Pre-specified

Adjusted Mean Change From Baseline in ppFEV1

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.

Time frame: Day 15, Day 33, Day 54

Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 150.06 percentage of predictedStandard Error 1.96
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 260.46 percentage of predictedStandard Error 2.64
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1Day 331.54 percentage of predictedStandard Error 2.4
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1Day 541.68 percentage of predictedStandard Error 2.88
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 152.50 percentage of predictedStandard Error 1.96
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1Day 54-1.63 percentage of predictedStandard Error 2.88
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 263.22 percentage of predictedStandard Error 2.64
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1Day 330.05 percentage of predictedStandard Error 2.4
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1Day 540.61 percentage of predictedStandard Error 2.88
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 26-0.98 percentage of predictedStandard Error 2.64
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1Day 33-2.17 percentage of predictedStandard Error 2.4
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 15-4.03 percentage of predictedStandard Error 1.96
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 15-1.85 percentage of predictedStandard Error 2.14
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 26-1.39 percentage of predictedStandard Error 2.89
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1Day 54-2.43 percentage of predictedStandard Error 3.15
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1Day 33-3.21 percentage of predictedStandard Error 2.63
Placebo SADAdjusted Mean Change From Baseline in ppFEV1Day 54-2.58 percentage of predictedStandard Error 2.49
Placebo SADAdjusted Mean Change From Baseline in ppFEV1Day 33-2.70 percentage of predictedStandard Error 2.08
Placebo SADAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 26-0.80 percentage of predictedStandard Error 2.28
Placebo SADAdjusted Mean Change From Baseline in ppFEV1(pre-dose) Day 15-0.83 percentage of predictedStandard Error 1.7
Other Pre-specified

Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo

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.

Time frame: Day 15, Day 33, Day 54

Population: All subjects treated with either QR-010 or placebo in the Multiple Ascending Dose cohorts that received at least 10 out of 12 doses.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 150.89 percentage of predictedStandard Error 2.59
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 334.24 percentage of predictedStandard Error 3.18
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 261.26 percentage of predictedStandard Error 3.49
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 544.26 percentage of predictedStandard Error 3.81
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 264.02 percentage of predictedStandard Error 3.49
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 153.32 percentage of predictedStandard Error 2.59
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 540.96 percentage of predictedStandard Error 3.8
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 332.75 percentage of predictedStandard Error 3.18
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 26-0.17 percentage of predictedStandard Error 3.49
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 15-3.20 percentage of predictedStandard Error 2.59
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 330.53 percentage of predictedStandard Error 3.18
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 543.20 percentage of predictedStandard Error 3.81
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 540.15 percentage of predictedStandard Error 4.01
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 33-0.51 percentage of predictedStandard Error 3.35
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 26-0.59 percentage of predictedStandard Error 3.68
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to PlaceboDay 15-1.02 percentage of predictedStandard Error 2.73
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline ppFEV1 as outcome variable and including treatment, baseline ppFEV1 value, time and interaction between time and treatment as covariatep-value: 0.193895% CI: [-2.3, 10.79]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline ppFEV1 as outcome variable and including treatment, baseline ppFEV1 value, time and interaction between time and treatment as covariates.p-value: 0.394395% CI: [-3.79, 9.29]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline ppFEV1 as outcome variable and including treatment, baseline ppFEV1 value, time and interaction between time and treatment as covariates.p-value: 0.868895% CI: [-6.01, 7.07]Mixed Models Analysis
Comparison: A mixed-model analysis was performed with repeated time measures on the change from baseline ppFEV1 as outcome variable and including treatment, baseline ppFEV1 value, time and interaction between time and treatment as covariates.p-value: 0.881395% CI: [-7.41, 6.39]Mixed Models Analysis
Other Pre-specified

Adjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup 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.

Time frame: Day 15, Day 33, Day 54

Population: 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.

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 153.11 percentage of predictedStandard Error 2.99
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 3310.19 percentage of predictedStandard Error 4.22
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 267.97 percentage of predictedStandard Error 5.39
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 157.93 percentage of predictedStandard Error 2.76
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 337.99 percentage of predictedStandard Error 3.91
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 2610.90 percentage of predictedStandard Error 4.98
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 264.65 percentage of predictedStandard Error 4.71
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 15-0.05 percentage of predictedStandard Error 2.59
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 333.50 percentage of predictedStandard Error 3.69
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 152.38 percentage of predictedStandard Error 2.74
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 333.21 percentage of predictedStandard Error 3.89
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 as Compared to Placebo (Subgroup ppFEV1 <90% at Baseline)Day 263.65 percentage of predictedStandard Error 4.97
p-value: 0.030195% CI: [1.13, 19.25]Mixed Models Analysis
p-value: 0.060195% CI: [-0.39, 16.37]Mixed Models Analysis
p-value: 0.35895% CI: [-4.4, 11.41]Mixed Models Analysis
p-value: 0.422495% CI: [-5.13, 11.55]Mixed Models Analysis
Other Pre-specified

Adjusted Mean Change From Baseline in ppFEV1 (Subgroup 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.

Time frame: Day 15, Day 33, Day 54

Population: 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..

ArmMeasureGroupValue (MEAN)Dispersion
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 15-0.29 percentage of predictedStandard Error 2.22
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 336.01 percentage of predictedStandard Error 3.16
QR-010 SAD 6.25 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 264.21 percentage of predictedStandard Error 4.05
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 267.15 percentage of predictedStandard Error 3.51
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 154.54 percentage of predictedStandard Error 1.92
QR-010 SAD 12.5 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 333.81 percentage of predictedStandard Error 2.74
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 260.89 percentage of predictedStandard Error 3.13
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 15-3.45 percentage of predictedStandard Error 1.71
QR-010 SAD 25 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 33-0.68 percentage of predictedStandard Error 2.44
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 15-1.01 percentage of predictedStandard Error 1.91
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 33-0.97 percentage of predictedStandard Error 2.73
QR-010 SAD 50 mgAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 26-0.10 percentage of predictedStandard Error 3.5
Placebo SADAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 26-3.76 percentage of predictedStandard Error 3.52
Placebo SADAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 15-3.40 percentage of predictedStandard Error 1.95
Placebo SADAdjusted Mean Change From Baseline in ppFEV1 (Subgroup ppFEV1 <90% at Baseline)Day 33-4.18 percentage of predictedStandard Error 2.76

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026