Status:
WITHDRAWN
Safety and Tolerability Study With VY-HTT01, in Adults With Early Manifesting Huntington's Disease
Lead Sponsor:
Voyager Therapeutics
Conditions:
Huntington Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief 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. ...
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...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
July 30 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 30 2024
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04885114
Start Date
July 30 2021
End Date
December 30 2024
Last Update
August 16 2021
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