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: 06 Jul 2018

At a glance

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

    • 05 Jun 2018 According to an Audentes Therapeutics media release, based on the data of this study, the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM).
    • 16 May 2018 According to an Audentes Therapeutics media release, the company is completing the analysis of the muscle biopsies from initial cohort 1 patients. These data, along with the safety and efficacy assessments from all patients, will be reviewed with the independent Data Monitoring Committee which is expected in the third quarter of 2018. The review is to make a decisions regarding dose escalation of the study.
    • 16 May 2018 Interim results presented in an Audentes Therapeutics media release.
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