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

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.

NCT Number	NCT05243017
Lead Sponsor	UniQure Biopharma B.V.
Conditions	Huntington Disease
Enrollment	14 participants
Start Date	2021-10-07
Primary Completion	2029-03 (estimated)
Study Completion	2029-10-07 (estimated)
Updated on ClinicalTrials.gov	2025-03-10