| Clinical trials identifier | NCT02564354 |
|---|---|
| Study codes | PQ-010-002 |
| Study title | Study 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 |
| Sponsor | Pro-QR |
| Drug (brand name) | QR-010 (Single-stranded RNA antisense oligonucleotide in iso-osmolar solution) |
| Drug administration | Intranasal administration of atomized liquid |
| Eligible CF patients | People 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 timeframe | The 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: https://proqr.gcs-web.com/news-releases/news-release-details/proqr-announces-qr-010-meets-primary-endpoint-proof-concept and from clinicaltrials.gov. |