Dose Escalation Study of QR-010 in Homozygous ?F508 Cystic Fibrosis Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02532764
• Date of registration: 13/08/2015
• Prospective Registration: No
• Primary sponsor: ProQR Therapeutics
• Public title: Dose Escalation Study of QR-010 in Homozygous ?F508 Cystic Fibrosis Patients
• Scientific title: Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ?F508 Cystic Fibrosis
• Date of first enrolment: June 2015
• Target sample size: 70
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02532764
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
• Phase: Phase 1/Phase 2

Countries of recruitment

Belgium, Canada, Czech Republic, Czechia, Denmark, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: Stuart Elborn, MD
• Affiliation: Trust and Queen's University Belfast

Key inclusion & exclusion 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 ?F508 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



Age minimum: 18 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: QR-010

Primary Outcome(s)

Severity of Treatment Emergent Adverse Events From Baseline Through End of Study [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit. [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Secondary Outcome(s)

Serum Clearance (CL) [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings. [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Area Under the Curve to Infinity [AUC(0-8)] [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Terminal Half-life (T1/2) [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Time to Maximum Serum Concentration [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Area Under the Curve to Final Sample [AUC(0-last)] [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Maximum Serum Concentration [Time Frame: 8 Days for Single-dose cohorts; 8 weeks for Multiple-dose cohorts]

Secondary ID(s)

PQ-010-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

European Commission

Results

• Results available: Yes
• Date Posted: 06/02/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02532764