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An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age With Leber Congenital Amaurosis Type 10 (LCA10) Due to the c.2991 +1655A>G (p.Cys998X) Mutation

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Trial Profile

An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age With Leber Congenital Amaurosis Type 10 (LCA10) Due to the c.2991 +1655A>G (p.Cys998X) Mutation

Status: Recruiting
Phase of Trial: Phase II/III

Latest Information Update: 06 Mar 2024

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At a glance

  • Drugs Sepofarsen (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions
  • Acronyms BRIGHTEN
  • Sponsors ProQR Therapeutics

    • Most Recent Events

    • 13 Apr 2022 According to a ProQR Therapeutics media release, bsed on the recommendation of the Data Safety and Monitoring Committee (DSMC), the Company currently plans to continue this study, until further regulatory guidance, after which next steps will be determined.
    • 18 Nov 2021 According to a ProQR Therapeutics media release, enrollment of 5 patients in the open-label, dose-escalation portion of this study is complete, with the double masked randomization portion of the trial getting underway.
    • 18 Nov 2021 According to a ProQR Therapeutics media release, the company present data of this study at the Analyst Event via webcast.

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