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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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Trial Profile

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: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 11 Jun 2025

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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

  • 17 Apr 2025 According to an uniQure media release, based on the data from this and NCT04120493 studies, US FDA has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington's disease.
  • 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.
  • 27 Feb 2025 According to an uniQure media release, the company completed patient enrollment in the third cohort of this trial and the company expects to provide an initial safety update on the third cohort in the second quarter of 2025.

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