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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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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: Not yet recruiting
Phase of Trial: Phase III

Latest Information Update: 27 Mar 2025

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At a glance

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

    • Most Recent Events

    • 27 Mar 2025 New trial record

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