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Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease

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Trial Profile

Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease

Status: Suspended
Phase of Trial: Phase II

Latest Information Update: 29 May 2025

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At a glance

  • Drugs RP A501 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Pharmacodynamics; Registrational
  • Sponsors Rocket Pharmaceuticals

Most Recent Events

  • 27 May 2025 Status changed from active, no longer recruiting to suspended, According to a Rocket Pharmaceuticals media release.
  • 27 May 2025 According to a Rocket Pharmaceuticals media release, A patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected Serious Adverse Event (SAE). company voluntarily paused further dosing in the study. On May 23, 2025, the FDA placed a clinical hold on the trial to allow for further evaluation.
  • 08 May 2025 According to a Rocket Pharmaceuticals media release, update expected mid-year 2025 and clinical data readout expected in mid-year 2026

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