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

NCT04885114 · Status: WITHDRAWN · Phase: PHASE1 · Type: INTERVENTIONAL

Last updated 2021-08-16

No results posted yet for this study

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.

Conditions

  • Huntington Disease

Interventions

GENETIC

Intraparenchymal rAAV1 - (mi)RNA HTT

Single dose MRI guided intraparenchymal infusion of rAAV1 - (mi)RNA HTT.

Sponsors & Collaborators

  • Voyager Therapeutics

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-30
Primary Completion
2024-12-30
Completion
2024-12-30
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04885114 on ClinicalTrials.gov