Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

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

A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.

The purpose of this study is to evaluate the safety, tolerability, and to determine the pharmacokinetics of QR-010 administered via inhalation in adult homozygous for ΔF508 Cystic Fibrosis.

Inclusion Criteria:

• Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of > 60 mmol/L
• Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation
• Body mass index (BMI) ≥ 17 kg/m2
• Non-smoking for a minimum of two years
• FEV1 ≥70% of predicted normal for age, gender, and height, at Screening
• Stable lung function
• Adequate hepatic and renal function

Exclusion Criteria:

• Breast-feeding or pregnant
• Use of lumacaftor or ivacaftor
• Use of any investigational drug or device
• History of lung transplantation
• Hemoptysis