Study 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.
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
1 other identifier
interventional
N/A
0 countries
N/A
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. This study is a first in human study, Phase 1b, open-label, randomized, multicenter, dose escalation study with a delayed treatment control arm.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
Started Jul 2021
Typical duration for phase_1
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 2, 2021
CompletedFirst Posted
Study publicly available on registry
May 13, 2021
CompletedStudy Start
First participant enrolled
July 30, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 30, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
December 30, 2024
CompletedAugust 16, 2021
August 1, 2021
3.4 years
March 2, 2021
August 9, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence and type of AEs
Safety will be assessed by measuring the number and type of AE or SAEs.
Collected for duration of study, average of 1 year after treatment
Secondary Outcomes (5)
Level of VY-HTT01 in blood
Collected for duration of study, average of 1 year after treatment
Unified Huntington Disease Rating Scale (UHDRS)
Collected for duration of study, average of 1 year after treatment
Clinical Global Impression (CGI) Measures
Collected for duration of study, average of 1 year after treatment
Huntington's Disease Quality of Life (HD-QOL) Measure
Collected for duration of study, average of 1 year after treatment
EuroQol 5 Dimension 5 Level (EQ-5D-5L) Measure
Collected for duration of study, average of 1 year after treatment
Other Outcomes (6)
Magnetic Resistance Imagining (MRI)
Collected for duration of study, average of 1 year after treatment
Levels of HTT protein in CSF
Collected for duration of study, average of 1 year after treatment
Levels of HTT protein in blood
Collected for duration of study, average of 1 year after treatment
- +3 more other outcomes
Study Arms (4)
Cohort 1 Unilateral low dose
EXPERIMENTAL3.0 x 10\^9 (vg/mL) rAAV1-miHHT
Cohort 2 Bilateral low dose
EXPERIMENTAL3.0 x 10\^9 (vg/mL) rAAV1-miHHT
Cohort 3 Bilateral mid dose
EXPERIMENTAL1.7 x 10\^10 (vg/mL) rAAV1-miHHT
Cohort 4 Bilateral high dose
EXPERIMENTAL9.9 x 10\^10 (vg/mL) rAAV1-miHHT
Interventions
Single dose MRI guided intraparenchymal infusion of rAAV1 - (mi)RNA HTT.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 2, 2021
First Posted
May 13, 2021
Study Start
July 30, 2021
Primary Completion
December 30, 2024
Study Completion
December 30, 2024
Last Updated
August 16, 2021
Record last verified: 2021-08