NCT02564354

Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects With CF With the ΔF508 CFTR Mutation

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: ProQR Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

Detailed description

This is an open-label, multi-center, exploratory study to estimate the effect of intranasal administration of QR-010 on the nasal mucosa in the restoration of CFTR function, as measured by nasal potential difference (NPD), in the nasal epithelium of adult subjects with CF who are homozygous or compound heterozygous for the ΔF508 CFTR mutation.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	QR-010	Single-stranded RNA antisense oligonucleotide in isoosmolar solution

Timeline

• Start date: 2015-09-01
• Primary completion: 2016-09-01
• Completion: 2016-09-01
• First posted: 2015-09-30
• Last updated: 2020-09-24
• Results posted: 2020-09-24

Locations

5 sites across 3 countries: United States, Belgium, France