ProQR Therapeutics recently announced the beginning of enrollment in the PQ-010-001 study, a global Phase 1b clinical study for the novel investigational RNA therapeutic QR-010, which is designed to repair the genetic mutation in the mRNA of those suffering with cystic fibrosis caused by the DF508 mutation.
“We are proud to announce that our first clinical study of QR-010 is now open and actively enrolling. Since the foundation of the company 3 years ago our team has worked very hard towards this step in the development of a therapy for CF, and we are excited to have reached this important milestone,” said Daniel de Boer, ProQR’s Chief Executive Officer.
“QR-010 is an innovative approach to target the underlying defect of CF. We are very pleased to participate in the first clinical trial for this novel compound,” explained Professor Stuart Elborn from Queen’s University Belfast who is also the past-President of the European Cystic Fibrosis Society.
PQ-010-001 is a randomized, placebo-controlled, double-blind Phase 1b trial that is a 28-day assessment to be conducted in 20 centers worldwide. This study will serve to evaluate the tolerability, safety and pharmacokinetics of both single and multiple ascending doses of inhaled QR-010 in 64 cystic fibrosis patients that are homozygotes for the trait and who carry 2 copies of the DF508 mutation. This study will also assess weight gain, sweat chloride, lung function and CFQ-R Respiratory Symptom Score. QR-010 will be administered to patients through inhalation for up a maximum of 3 times per week and up to 4 weeks. This trial will be carried out in parallel with a Nasal Potential Difference (NPD) evaluation that will start enrolling 16 cystic fibrosis patients that are either homo- or heterozygous for the DF508 mutation in third quarter of this year.
“The Phase 1b study and the NPD proof-of-concept study will provide a strong, early signal as to the therapeutic potential of QR-010,” noted Noreen Henig, MD, the ProQR’s Chief Development Officer.
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