uniQure

UniQure is active, but no longer recruitingSafety and Proof-of-Concept (POC) Study with AMT-130 in Adults with Early Manifest Huntington’s Disease

Find this study on clinicaltrials.gov and the uniQure website.

SponsoruniQure
The StudyTo test a new experimental gene therapy called AMT-130 in patients with early manifest (mild symptoms) HD in order to lower the huntingtin protein levels in the brain, which could help slow the progression of HD. The therapy works by using a virus to deliver a tiny piece of genetic material to target and reduce both normal and mutant huntingtin protein levels.

The trial has different groups of participants, with some receiving a low or high dose of AMT-130 and another group exploring whether combining the therapy with immune-suppressing treatment can improve safety. The therapy is delivered in a one-time surgical procedure where AMT-130 is injected directly into specific areas of the brain, and participants are monitored for up to five years.
PhaseI/II