Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis

• Status: Completed
• Trial end date: 2018-04-24
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

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.)