Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease (NCT04885114) | Clinical Trial Compass
WithdrawnPhase 1
Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease
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.
0Started 2021-07-30
Plain-language 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Must be at least 18 years old.
* Have CAGn repeat \>39.
* Have diagnostic confidence score of 4 based on motor, cognitive, or behavioral symptoms.
* Have a TFC score of 13 to 11.
* Have stable dosing of neurological and psychiatric medications.
* Capable of giving informed consent.
* Able to comply with all procedures and study visits.
Exclusion Criteria:
* Have any significant structural or degenerative neurologic disease other than HD.
* Have any chronic disability, significant systemic illness and/or, unstable medical condition, or clinical findings noted.
* Have primary or secondary immune-compromise due to infections or medical conditions or chronic therapies.
* Have contraindications to lumbar puncture or increased risks of bleeding upon surgery.
* Started or changed dose of a concomitant CNS medication within 30 days.
* Had prior neurosurgical procedures that could complicate the study procedures.
* Have used any investigational therapies within 30 days prior to Screening, oligonucleotide therapies within 9 months prior to Baseline, or any prior gene therapy.
* Male or female with reproductive capacity and is unwilling to use highly effective contraception for 12 months after surgery.
* Have contraindications to MRI such as claustrophobia, embedded metal in the body, or known allergy or intolerance to contrast agents.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence and type of AEs
Timeframe: Collected for duration of study, average of 1 year after treatment