Clinical trial NCT02566044: Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis
Official title: A Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Doses of Inhaled QBW276 in Patients With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Pulmonary Cystic Fibrosis
Keywords: Cystic Fibrosis
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2015-10-01
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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.


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.)

Primary outcomes:
measure: Cohorts 1 and 2: Safety Assessments, Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]).
timeFrame: Cohort 1: day 1-7; Cohort 2: day 1-14
measure: Cohorts 1 and 2: Pharmacokinetics (Cmax) of QBW276, QBP545, and QBV697 in Plasma
timeFrame: Cohort 1: day 1, 7; Cohort 2: day 1, 14
measure: Cohorts 1 and 2: Pharmacokinetics (Tmax) of QBW276, QBP545, and QBV697 in Plasma
timeFrame: Day 1, 7 and 14
measure: Cohorts 1 and 2: Pharmacokinetics (AUCtau) of QBW276, QBP545, and QBV697 in Plasma
timeFrame: Day 1, 7 and 14
measure: Cohorts 1 and 2: Pharmacokinetics Accumulation Ratio (Racc) of QBW276, QBP545, and QBV697 in Plasma
timeFrame: Cohort 1: 7 days; Cohort 2: 14 days
Secondary outcomes:
measure: Cohorts 1 and 2: Change From Baseline in Percent Predicted Forced Expiratory Volume in the First Second by Spirometry (% Predicted FEV1)
timeFrame: Baseline to End of study (EOS)
measure: Cohorts 1, 2: Change From Baseline in Lung Clearance Index (LCI) From Baseline to Day 7 for Cohort 1, Day 14 for Cohort 2.
timeFrame: Baseline to EOS
Other outcomes:
Locations:
city: New York
country: United States
facility: Novartis Investigative Site
state: New York
city: Dallas
country: United States
facility: Novartis Investigative Site
state: Texas
city: Essen
country: Germany
facility: Novartis Investigative Site
city: Koeln
country: Germany
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: