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

• Status: Completed
• Trial end date: 2016-09-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ProQR Therapeutics
• Collaborator: European Commission

Criteria

Inclusion Criteria:

• Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of > 60 mmol/L

• Nasal potential difference (NPD) measurement at Screening consistent with CF

• Confirmation of CFTR gene mutations homozygous or compound heterozygous for the ΔF508 mutation

• Body mass index (BMI) of ≥ 18 kg/m2

• Non-smoking for a minimum of 2 years

• Stable lung function

• FEV1 ≥40% of predicted normal for age, gender, and height at Screening

Exclusion Criteria:

• Breast-feeding or pregnant

• Acute allergy or infection affecting nasal conditions not resolved within 14 days prior Screening

• Use of lumacaftor or ivacaftor

• Use of any investigational drug or device

• Hemoptysis