Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ?F508 CFTR Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02564354
• Date of registration: 29/09/2015
• Prospective Registration: No
• Primary sponsor: ProQR Therapeutics
• Public title: Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ?F508 CFTR Mutation
• Scientific title: Open-Label, Exploratory Study to Evaluate the Effects of QR-010 on Nasal Potential Difference in Subjects With CF With the ?F508 CFTR Mutation
• Date of first enrolment: September 2015
• Target sample size: 18
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02564354
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Single (Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

Belgium, France, United States

Contacts

• Name: John P Clancy, MD
• Affiliation: Cincinnati Childrens Hospital Medical Center

Key inclusion & exclusion criteria

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 ?F508
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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: QR-010

Primary Outcome(s)

Intra-subject Change From Baseline of CFTR-mediated Total Chloride Transport as Measured by Nasal Potential Difference (NPD). [Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment.]

Secondary Outcome(s)

The Mean Change in CFTR-mediated Total Chloride Transport. [Time Frame: 2 and 4 weeks, and at 3 weeks post-treatment.]

Changes in Nasal Symptoms (Sino-Nasal Outcome Test - SNOT-22) From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Intra-subject Change of Sodium Transport as Measured by Nasal Potential Difference (NPD) From Baseline Through End of Study. [Time Frame: Baseline, at 2 and 4 weeks, and at 3 weeks post-treatment.]

Number of Subjects With a -4 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. [Time Frame: 2 and 4 weeks, and at 3 weeks post-treatment.]

Number of Subjects With Abnormalities of Physical Examinations From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Number of Subjects Experiencing Serious Adverse Events From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Number of Subjects With Abnormalities of Vital Signs & Oximetry From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Number of Subjects With Abnormalities of Laboratory Parameters From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Changes in Nasal Symptoms (Based on the Nasal Examination Rating Scale - NERS) From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Number of Subject Discontinuations Due to AEs From Baseline Through End of Study. [Time Frame: 3 weeks post-treatment.]

Number of Subjects With a -6.6 mV or More Negative Change in CFTR-mediated Total Chloride Transport, and After Different Treatment Durations From Baseline Through End of Study. [Time Frame: 2 and 4 weeks, and at 3 weeks post-treatment.]

Secondary ID(s)

PQ-010-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

European Commission

Results

• Results available: Yes
• Date Posted: 24/09/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02564354