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A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene

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Trial Profile

A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 20 Jun 2025

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At a glance

  • Drugs Sepofarsen (Primary)
  • Indications Leber congenital amaurosis
  • Focus Registrational; Therapeutic Use
  • Acronyms HYPERION
  • Sponsors Laboratoires Thea

Most Recent Events

  • 15 Jun 2025 Planned initiation date changed from 1 Mar 2025 to 1 Aug 2025.
  • 15 Jun 2025 Status changed from not yet recruiting to recruiting.
  • 27 Mar 2025 New trial record

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