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

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.

Timeline

Start

2021-07-30

Primary completion

2024-12-30

Completion

2024-12-30

Outcome

Outcome not reported

Stopped: “We have discontinued our 1st generation HD program with the VYTAL Study \& have initiated a 2nd generation program using a novel, proprietary AAV capsid that may enable intravenous administration \& achieve widespread distribution to affected tissue.”

Drugs

Indications