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ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients

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Trial Profile

ASPIRO: A Phase 1/2/3, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Efficacy of AT132, an AAV8-Delivered Gene Therapy in X-Linked Myotubular Myopathy (XLMTM) Patients

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 01 Feb 2024

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At a glance

  • Drugs Resamirigene bilparvovec (Primary)
  • Indications Congenital structural myopathies; X-linked genetic disorders
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Acronyms ASPIRO
  • Sponsors Astellas Gene Therapies

    • Most Recent Events

    • 19 Dec 2023 Planned End Date changed from 31 Oct 2030 to 31 Mar 2030.
    • 01 Dec 2023 Results published in the Lancet Neurology
    • 17 Nov 2023 Results published in the Genethon Media Release
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