NCT04885114

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

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• Status: Withdrawn
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Voyager Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Huntington Disease

Interventions

Type	Name	Description
GENETIC	Intraparenchymal rAAV1 - (mi)RNA HTT	Single dose MRI guided intraparenchymal infusion of rAAV1 - (mi)RNA HTT.

Timeline

• Start date: 2021-07-30
• Primary completion: 2024-12-30
• Completion: 2024-12-30
• First posted: 2021-05-13
• Last updated: 2021-08-16

Regulatory

• FDA-regulated drug study