Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease

Huntington Disease Clinical Trial

Official title: A Phase 1B, Open-label, Randomized, Controlled, Multicenter, Dose Escalation Study of the Safety, Tolerability, and Biological Effects of VY-HTT01 Administered Via Intraparenchymal Infusion of the Putamen and Thalamus in Adults With Huntington's Disease

Status Withdrawn

Clinical Trial Summary

This is the first clinical study of VY-HTT01, a gene therapy for early-stage Huntington's Disease (HD) patients. The primary goal of this trial is to evaluate the safety and tolerability of VY-HTT01. This study is a first in human study, Phase 1b, open-label, randomized, multicenter, dose escalation study with a delayed treatment control arm.

Clinical Trial Description

This dose escalation trial will evaluate the safety and tolerability of 4 single dose levels of VY-HTT01. The maximum duration that a subject randomized to treatment may be involved in the study is up to 15 months. Delayed treatment subjects will be followed for a minimum of 6 months as a control before moving up into the treatment arm in the next cohort. The maximum duration that a delayed treatment subject may be involved in the study is up to 24 months. Subjects who participate in this study will be asked to enroll in a long-term observation study. ;

Related Conditions & MeSH terms

• Huntington Disease

• NCT number: NCT04885114
• Study type: Interventional
• Source: Voyager Therapeutics
• Status: Withdrawn
• Phase: Phase 1
• Start date: July 30, 2021
• Completion date: December 30, 2024