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Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease

NCT05243017 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2025-03-10

No results posted yet for this study

Summary

This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration.

Cohort 3 participants will receive either high or low dose (1:1 randomization). Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab.

Conditions

  • Huntington Disease

Interventions

GENETIC

intra-striatal rAAV5-miHTT

One time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain

Sponsors & Collaborators

  • UniQure Biopharma B.V.

    lead INDUSTRY

Principal Investigators

  • David Margolin, MD, PhD · UniQure Biopharma B.V.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

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

Timeline & Regulatory

Start
2021-10-07
Primary Completion
2029-03-31
Completion
2029-10-07

Countries

  • Poland
  • United Kingdom

Study Locations

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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 NCT05243017 on ClinicalTrials.gov