Date: 2017-04-27
Type of information: Initiation of the trial
phase: 1-2
Announcement: clinical trial authorization
Company: ProQR Therapeutics (The Netherlands)
Product: QR-110
Action mechanism: antisense oligonucleotide/RNA. QR-110 is a novel investigational RNA therapeutic targeting LCA 10 due to the p.Cys998X mutation, a severe genetic rare disease that causes children to lose sight in the first years of life. The goal of QR-110 is to repair the underlying defect in the RNA, such that it leads to a normal “wild-type” mRNA. The RNA is the "blueprint" for protein synthesis. By repairing this "blueprint" a normal healthy protein will be formed that is expected to have normal function.
Disease: Leber’s congenital amaurosis type 10
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Country: Belgium, USA
Trial details: PQ-110-001 is an open-label trial that will include approximately 6 children (age 6- 17 years) and 6 adults (? 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; one every three months for one year. The eye that does not receive injections is the control. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe. The primary endpoints will be safety and tolerability. Secondary efficacy endpoints will assess the pharmacokinetics and restoration/improvement of visual function and retinal structure through ophthalmic tests such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in LCA subjects will also be evaluated. Top-line results from the trial are expected to be available in 2018. (NCT03140969)
Latest news: • On April 27, 2017, ProQR Therapeutics announced that the company can start the Phase 1/2 trial for QR-110, named PQ-110-001, in Leber’s congenital amaurosis Type 10 (LCA 10) patients. LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available.
