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

• Status: Completed
• Trial end date: 2017-09-14
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1/Phase 2
• 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

• 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