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Clinical trials identifierNCT02190604
Study codesCQBW251X2101
Study titleA Randomized, Double Blind Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Preliminary Pharmacodynamics of Single and Multiple Ascending Doses of QBW251 in Healthy Subjects and Multiple Doses in Cystic Fibrosis Patients
SponsorNovartis Pharmaceuticals
Drug (brand name)QBW251
Drug administrationOral capsule
Eligible CF patientsAged at least 18 years with at least one CFTR mutation in Class II, IV, V, VI, or with 2 copies of the F508del CFTR mutation (Part 3 only)
Controlled study?Yes
Randomised study?Yes
Blinded study?Yes (triple blind - neither medical staff, nor participants, nor Investigator knew which treatment participants were taking)
Extension study?No
Study timeframeThe study was conducted between July 2012 and November 2015




Study:This Phase 1/2 study was conducted in 23 sites across 7 countries.
Aim:To assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics (proof of concept) of QBW251 in healthy subjects and CF patients following single and multiple doses.
Parts 1 and 2 of this study were performed in healthy volunteers.
Parts 3 and 4 were performed in people with CF.
Participants:153 participants were enrolled (104 healthy volunteers and 49 people with CF). There were 23 participants from 11 ECFS-CTN sites in France, Germany and the UK (Montpellier, Lyon, Cochin, Berlin, Koln, Belfast, Leeds, London). 
Treatment:

In Part 2, healthy volunteers received placebo or a single dose of QBW251 (between 10 mg and 1000 mg).
In Part 2, healthy volunteers received multiple doses of placebo or QBW251 (between 150 mg and 750 mg) over a 14-day treatment period.
In Part 3, people with CF received multiple doses of placebo or QBW251 (150 mg or 450 mg) over a 14-day treatment period.

Safety:    In healthy volunteers (Parts 1 and 2), the rate of side effects was comparable between placebo and QBW251 groups. There were no serious adverse events.
In Part 3, most people with CF had adverse events. One patient in each QBW251 group had a serious adverse event.
Efficacy:In people with CF, the change in lung clearance index (LCI) was measured between baseline and Day 15. LCI improved slightly in patients on QBW251 450 mg but this was not clinically significant. This study was terminated early, before Part 4 of the study was initiated in people with CF, as planned in the study protocol. The sponsor did not provide a reason for this early termination.
Data source:The summary of data provided here is from clinicaltrials.gov.


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