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Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Trial Profile

Double-masked, Randomized, Controlled, Multiple-dose Study to Evaluate Efficacy, Safety, Tolerability and Syst. Exposure of QR-110 in Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Status: Recruiting
Phase of Trial: Phase II/III

Latest Information Update: 06 Nov 2019

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At a glance

  • Drugs QR 110 (Primary)
  • Indications Leber congenital amaurosis
  • Focus Registrational; Therapeutic Use
  • Acronyms ILLUMINATE
  • Sponsors ProQR Therapeutics
  • Most Recent Events

    • 06 Nov 2019 According to a ProQR Therapeutics media release, top-line data from this study is expected during the first half of 2021.
    • 10 Oct 2019 According to a ProQR Therapeutics media release, top-line data from Phase 1/2 (PQ-110-001) study of LCA10 strengthen confidence in the design of this trial which could be the sole registration trial for the sepofarsen program.
    • 15 Apr 2019 According to a ProQR Therapeutics media release, first patient has been dosed in this study and results are expected at year-end 2020.
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