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A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

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Trial Profile

A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 13 Nov 2024

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At a glance

  • Drugs RP-A601 (Primary)
  • Indications Cardiomyopathies
  • Focus Adverse reactions
  • Sponsors Rocket Pharmaceuticals

    • Most Recent Events

    • 07 Nov 2024 According to a Rocket Pharmaceuticals media release, enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM. Preliminary data expected in the first half of 2025.
    • 02 Sep 2024 Trial design of this trial has been presented at the ESC Congress 2024 - Annual Congress of the European Society of Cardiology
    • 09 Oct 2023 Planned End Date changed from 1 May 2026 to 1 Sep 2026.

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