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A Phase Ib/II Randomized, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease

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A Phase Ib/II Randomized, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 13 Sep 2024

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At a glance

  • Drugs AMT-130 (Primary)
  • Indications Huntington's disease
  • Focus Adverse reactions; Proof of concept; Therapeutic Use
  • Sponsors uniQure

    • Most Recent Events

    • 09 Jul 2024 According to an uniQure media release, In the second half of 2024, uniQure expects to hold a Type B, multi-disciplinary RMAT meeting with U.S. Food and Drug Administration (FDA) to present the updated data and discuss potential expedited clinical development pathways and accelerated approval.
    • 09 Jul 2024 According to an uniQure media release, In mid-2025, uniQure expects to present another interim analysis from the ongoing Phase I/II studies of AMT-130. The data will include a 36-month comparison of treated patients to the propensity score-weighted external control.
    • 09 Jul 2024 According to an uniQure media release, company continues accumulating longer-term patient outcomes from the Phase I/II studies to support the emerging therapeutic benefit and look forward to holding an initial, multi-disciplinary RMAT meeting with the FDA later this year (in the second half of 2024) to discuss the potential for expedited clinical development of AMT-130.

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