NCT04120493

Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

A Phase 1/2, Randomized, Double-Blind, Sham Control and Open-Label Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington's Disease

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 43 (estimated)
• Sponsor: UniQure Biopharma B.V. · Industry
• Sex: All
• Age: 25 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.

Detailed description

AMT-130 is an investigational, single administration gene therapy intended to modify the disease course for HD. Preclinical studies have shown that AMT-130 lowers huntingtin protein and is associated with decreased progression of Huntington's Disease signs in animal models. Cohort 1, 2, and 3 evaluated low dose and high dose AMT-130. Cohort 4 will further evaluate the safety of high dose AMT-130 in participants with low striatal volume. All participants in Cohort 4 will receive high dose AMT-130 and will receive pre- and post-operative dexamethasone. Cohorts 1 and 2 participants continue follow-up visits through 6 years after receipt of AMT-130. Cohorts 3 and 4 participants continue follow-up visits through 5 years after receipt of AMT-130.

Conditions

• Huntington's Disease

Interventions

Type	Name	Description
GENETIC	intra-striatal rAAV5-miHTT	One time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain
OTHER	Imitation (sham) surgery	Simulated surgical procedure with skin incisions only; no intrastriatal injections and no burr holes through the skull

Timeline

• Start date: 2019-09-06
• Primary completion: 2029-06-01
• Completion: 2029-12-01
• First posted: 2019-10-09
• Last updated: 2025-10-21

Locations

12 sites across 1 country: United States

Regulatory

• FDA-regulated drug study