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



Status:Recruiting
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - Any
Updated:6/23/2016
Start Date:September 2015
End Date:December 2016
Contact:Nigel Tomkinson, RN
Email:ntomkinson@proqr.com

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Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects With CF With the ΔF508 CFTR Mutation

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).

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.

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
We found this trial at
4
sites
Leuven, 3001
Principal Investigator: Christiane De Boeck, MD
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1720 2nd Ave S
Birmingham, Alabama 35233
(205) 934-4011 
Principal Investigator: George Solomon, MD
University of Alabama at Birmingham The University of Alabama at Birmingham (UAB) traces its roots...
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Principal Investigator: John P Clancy, MD
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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1400 Jackson St
Denver, Colorado 80206
(303) 388-4461
National Jewish Health National Jewish Health is known worldwide for treatment of patients with respiratory,...
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Denver, CO
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