Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease

Huntington Disease Clinical Trial

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

Status Recruiting

Clinical Trial Summary

This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration.

Clinical Trial Description

The aim of the European study is to build upon the safety demonstrated in the first human dose (FHD) randomized, double blind, sham-controlled sequential dose escalation study (CT-AMT-130-01; clinicaltrials.gov NCT04120493) being conducted in the US and expand the number of patients exposed to the two doses to provide sufficient sample size for comparisons of safety and efficacy. CT-AMT-130-02 is a Phase Ib/II open-label sequential ascending dose study that will be conducted in approximately 5 to 8 European HD centers; 3 to 4 of these centers will serve as surgical sites. Both studies will share a common set of clinical, safety, imaging, and biomarker evaluations over 5 years of follow-up. The DSMB will evaluate safety and other parameters to enable the staggered treatment of patients within each of the dosing cohorts ;

Related Conditions & MeSH terms

• Huntington Disease

• NCT number: NCT05243017
• Study type: Interventional
• Source: UniQure Biopharma B.V.
• Contact: Diane Lopez, MS
• Phone: 781-777-3697
• Email: amt130_clinical_trials@uniqure.com
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 7, 2021
• Completion date: October 7, 2027