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

ProQR Therapeutics

Status and phase

Completed

Phase 1

Conditions

Cystic Fibrosis

Treatments

Drug: QR-010

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT02564354

PQ-010-002

Details and patient eligibility

About

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).

Full 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.

Enrollment

18 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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