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A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene

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A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene

Status: Recruiting
Phase of Trial: Phase II/III

Latest Information Update: 22 Dec 2024

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At a glance

  • Drugs Laruparetigene zovaparvovec (Primary)
  • Indications Retinitis pigmentosa
  • Focus Registrational; Therapeutic Use
  • Acronyms VISTA
  • Sponsors Applied Genetic Technologies Corporation; Beacon Therapeutics

    • Most Recent Events

    • 27 Mar 2024 Planned number of patients changed from 63 to 75.
    • 27 Mar 2024 Status changed from not yet recruiting to recruiting.
    • 27 Mar 2024 Planned End Date changed from 1 Mar 2029 to 1 Oct 2029.

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