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A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

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Trial Profile

A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 21 Jan 2025

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At a glance

  • Drugs GTX 102-GeneTx Biotherapeutics (Primary)
  • Indications Angelman syndrome
  • Focus Adverse reactions
  • Sponsors Ultragenyx Pharmaceutical

    • Most Recent Events

    • 15 Aug 2024 Status changed from not yet recruiting to recruiting.
    • 02 Aug 2024 According to an Ultragenyx Pharmaceutical media release, the company has also participated in a PRIME meeting with the European Medicines Agency, receiving acceptance of the overall Phase 3 study design, dosing and evaluations and has met with Japans Pharmaceuticals and Medical Devices Agency to inform and discuss its Phase 3 study design.
    • 02 Aug 2024 According to an Ultragenyx Pharmaceutical media release, the trial expected to initiate in end of the year.

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