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

Trial Profile

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

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 14 Nov 2017

At a glance

  • Drugs AT 132 (Primary)
  • Indications Congenital structural myopathies
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms ASPIRO
  • Sponsors Audentes Therapeutics
  • Most Recent Events

    • 14 Nov 2017 According to an Audentes Therapeutics media release, enrollment in the first dose cohort has been completed in this trial and the company plans to report preliminary data in early January 2018.
    • 11 Oct 2017 According to an Audentes Therapeutics media release, the primary efficacy analysis is expected to be conducted at 12 months, with interim evaluations expected to be conducted at earlier time points. After the primary 12-month assessment, subjects are expected to be followed for another four years to assess long term safety, durability of effect and developmental progression.
    • 03 Oct 2017 According to an Audentes Therapeutics media release, the Medicines and Healthcare Products Regulatory Agency (MHRA) has approved the Clinical Trial Authorisation (CTA) application and allow to enroll patients in the UK.
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