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An open-label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects of Age 3 to <8 Years 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, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects of Age 3 to <8 Years 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: 15 Sep 2023

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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

    • 17 Nov 2022 This trial has been completed in Belgium (Global end date 18 Oct 2022), according to European Clinical Trials Database record.
    • 01 Nov 2022 This trial has been completed in Germany (Global end date 18/10/2022), according to European Clinical Trials Database record.
    • 30 Oct 2022 Planned End Date changed to 13 Dec 2022.

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