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

Completed · Phase 1

Conditions studied: Cystic Fibrosis

In brief

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).

Key facts

Study ID

NCT02564354

Run by

ProQR Therapeutics

People needed

18

Starts

2015-09-01

Expected to finish

2016-09-01

Last updated by the study team

2020-09-24

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• National Jewish Health — Denver, Colorado, United States
• Cincinnati Childrens Hospital Medical Center — Cincinnati, Ohio, United States
• U.Z. Leuven — Leuven, Belgium
• Hopital Necker-Enfants Malades — Paris, France

Full record on ClinicalTrials.gov

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