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Clinical trials identifierNCT02532764
Study codesPQ-010-001
Study title    Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects with Homozygous ΔF508 Cystic Fibrosis
Drug (brand name)QR-010 (Single-stranded RNA antisense oligonucleotide in iso-osmolar solution)
Drug administrationInhalation
Eligible CF patientsPeople with CF aged at least 18 years, with 2 copies of the F508del CFTR mutation
Controlled study?Yes, placebo controlled
Randomised study?Yes
Blinded study?Yes
Extension study?No
Study timeframeThe study was conducted between June 2015 and September 2017

Study:This Phase 1b study was conducted in 27 sites across 9 countries. 
Aim:The aim was to determine the safety, tolerability, and pharmacokinetics of inhaled QR-010 administered via inhalation in adult with Cystic Fibrosis 2 copies of the ΔF508del CFTR mutation. 
Participants:64 people with CF were enrolled.
There were 51 participants from 16 ECFS-CTN sites in Belgium, Czech Republic, Denmark, France, Germany, Italy, Spain and the UK (Leuven, Prague, Copenhagen, Nantes, Lyon, Necker Paris, Berlin, Hannover, Munich, Rome, Verona, Barcelona, Belfast, London, Brussels, Southampton)
Treatment:Participants were randomised into 8 groups. Four groups received either a single dose of QR-10 (between 6.25 mg and 50 mg) and four groups received multiple doses of QR-10 (between 6.25 mg and 50 mg) 12 times over 4 weeks.
Safety:    Multiple doses of QR-10 were well tolerated with no serious adverse events judged to be related to QR-10. The most common adverse events were cough, fatigue and increased sputum.
Efficacy:This study was designed to primary assess safety and tolerability. The Cystic Fibrosis Questionnaire-Revised (CFQ-R), a validated patient reported outcome found that scores for the respiratory domain were better in the QR-10 groups versus the placebo group. This improvement is “minimally clinically significant”.
Data source:This preliminary summary of data is from a press release:, a conference poster, available here: and from