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An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

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Trial Profile

An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 18 Oct 2024

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At a glance

  • Drugs Sepofarsen (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions; First in man
  • Sponsors Laboratoires Thea; ProQR Therapeutics

Most Recent Events

  • 01 May 2022 Results assessing sepofarsen safety and tolerability via the frequency and severity of ocular adverse events (AEs) published in the Nature Medicine
  • 24 Sep 2020 According to an ProQR Therapeutics media release, data from this trial will be presented at the European Society of Retina Specialists (Euretina) congress.
  • 08 Jun 2020 According to an ProQR Therapeutics media release, data from this study will be shared via a video presentation through the Association for Research in Vision and Ophthalmology (ARVO). Due to COVID-19, the ARVO 2020 Annual Meeting was cancelled and instead video-recorded presentations will be available on ARVOLearn, starting June 15, 2020.

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