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

NCT04120493 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 43

Last updated 2025-10-21

No results posted yet for this study

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.

Conditions

Interventions

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

Sponsors & Collaborators

  • UniQure Biopharma B.V.

    lead INDUSTRY

Principal Investigators

  • David H. Margolin, MD, PhD · uniQure, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
25 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-06
Primary Completion
2029-06-30
Completion
2029-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

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 NCT04120493 on ClinicalTrials.gov