Clinical Trial

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

Active, Not Recruiting Phase 1/2
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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.
Protocol Amendment History 40 amendments
This ClinicalTrials.gov record has been amended 40 times since 2019-10-07; most recent amendment 2025-10-17.
Status change: Recruiting → Active, Not Recruiting 2025-10-17
Status change: Active, Not Recruiting → Recruiting 2025-08-11
Status change: Recruiting → Active, Not Recruiting 2025-03-05
Status change: Active, Not Recruiting → Recruiting 2024-12-18
Status change: Recruiting → Active, Not Recruiting 2024-09-30
Status change: Not Yet Recruiting → Recruiting 2019-10-11
Trial Details
NCT Number NCT04120493
Lead Sponsor UniQure Biopharma B.V.
Conditions Huntington's Disease
Enrollment 43 participants
Start Date 2019-09-06
Primary Completion 2029-06 (estimated)
Study Completion 2029-12 (estimated)
Updated on ClinicalTrials.gov 2025-10-21