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Clinical trials identifierNCT02564354
Study codesPQ-010-002
Study titleStudy title    Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects with CF with the ΔF508 CFTR Mutation
Drug (brand name)QR-010 (Single-stranded RNA antisense oligonucleotide in iso-osmolar solution)
Drug administrationIntranasal administration of atomized liquid
Eligible CF patientsPeople with CF aged at least 18 years, with 1 or 2 copies of the F508del CFTR mutation
Controlled study?No
Randomised study?No
Blinded study?No
Extension study?No
Study timeframeThe study was conducted between September 2015 and September 2016

Study:This Phase 1 study was conducted in 5 sites across 3 countries.
Aim:The aim was to determine whether intranasal administration of QR-010 increases the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). 
Participants:18 people with CF were enrolled (10 had 2 copies of the F508del CFTR mutation and 8 had 1 copy and another CF causing mutation).
There were 9 participants from 2 ECFS-CTN sites in France and Belgium (Leuven, Necker Paris).
Treatment:QR-10 was applied to the inside of the nose 12 times over 4 weeks. 
Safety:    Safety has not yet been reported.
Efficacy:CFTR activity was measured using nasal potential difference (NPD). Participants with 2 copies of the F508del CFTR mutation, and who adhered perfectly to the study conditions (7 patients in total), had improved NPD at Day 26 compared to baseline (-4.1 mV; p=0.0389). The change in NPD was not significant for patients with 1 copy of F508del and 1 copy of another CF causing mutation
Data source:This preliminary summary of data provided here is from a press release:
and from