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A Phase I/II, Randomized, Double-Blind, Sham Control 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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Trial Profile

A Phase I/II, Randomized, Double-Blind, Sham Control 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: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 09 Jun 2025

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At a glance

  • Drugs AMT-130 (Primary)
  • Indications Huntington's disease
  • Focus Adverse reactions; First in man; Proof of concept
  • Acronyms HD-GeneTRX-1
  • Sponsors uniQure

Most Recent Events

  • 17 Apr 2025 According to an uniQure media release, based on the data from this and NCT05243017 studies, The U.S FDA has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease.
  • 10 Mar 2025 Status changed from recruiting to active, no longer recruiting.
  • 27 Feb 2025 According to an uniQure media release, In the third quarter of 2025, the Company expects to present data from its ongoing Phase I/II studies of AMT-130 in support of a potential BLA submission. The update will include follow-up data on all patients treated with AMT-130 in the first two cohorts, including three years of follow-up on 24 treated patients.

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