Phase 2
Completed N=16
Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis
Pulmonary Cystic Fibrosis
Source: ClinicalTrials.gov NCT02566044 ↗
Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Jul 2019
Primary outcomePrimary: Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]). — 0; 0; 0; 0 Participants
Summary
This is a study of multiple doses of inhaled QBW276 in patients with cystic fibrosis on top of standard of care. The study was divided into 3 Cohorts. Cohorts 1 and 2 are designed to be a randomized, double-blind, placebo-controlled, parallel arm, multiple dose study to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of inhaled QBW276 over 1 week (cohort 1) or 2 weeks (cohort 2) in patients with cystic fibrosis regardless of their genotype.
The study was terminated after Cohort 2 due to the resource issues.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]). |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Cohorts 1 and 2: Pharmacokinetics (Cmax) of QBW276, QBP545, and QBV697 in Plasma |
0.159; 3.88; 1.21; 0.174; 5.98; 1.63 | — |
| PRIMARY Cohorts 1 and 2: Pharmacokinetics (Tmax) of QBW276, QBP545, and QBV697 in Plasma |
0.183; 0.375; 0.433; 0.250; 0.250; 0.250 | — |
| PRIMARY Cohorts 1 and 2: Pharmacokinetics (AUCtau) of QBW276, QBP545, and QBV697 in Plasma |
0.0332; 14.3; 1.35; 0.0634; 13.9; 1.79 | — |
| PRIMARY Cohorts 1 and 2: Pharmacokinetics Accumulation Ratio (Racc) of QBW276, QBP545, and QBV697 in Plasma |
1.20; 1.28; 1.84; 1.59; 1.01; 1.74 | — |
| SECONDARY Cohorts 1 and 2: Change From Baseline in Percent Predicted Forced Expiratory Volume in the First Second by Spirometry (% Predicted FEV1) |
-0.1; -0.1; -2.7 | — |
| SECONDARY Cohorts 1, 2: Change From Baseline in Lung Clearance Index (LCI) From Baseline to Day 7 for Cohort 1, Day 14 for Cohort 2. |
1.530; 2.478; 0.470 | — |
Eligibility Criteria
Inclusion Criteria
- Cohorts 1 and 2 = any genotype on any standard of care treatment
- Cohort 3 = F508del homozygotes on standard of care at that time
- FEV₁between 40 and 100%
- LCI2.5 ≥ 8 if FEV₁is more than 80%
Exclusion Criteria
- Adrenal or electrolyte abnormalities
- Lung transplant
- Autonomic dysfunction (e.g. recurrent episodes of fainting, palpitations, etc.)
Data sourced from ClinicalTrials.gov (NCT02566044). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.