Clinical Trials

uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington’s disease. 

In the U.S. study, a total of 26 patients with early manifest Huntington’s disease were randomized to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) procedure (n=10). Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment. The European open-label Phase 1b/2 study of AMT-130 enrolled 13 patients with early manifest Huntington’s disease (n=6 low dose; n=7 high dose). 

A third cohort enrolled an additional 12 patients across sites in the U.S. and EU. This cohort was randomized to explore both doses of AMT-130 in combination with immunosuppression, using the current, established stereotactic administration procedure. 

We’ve initiated a fourth U.S. based cohort, evaluating high-dose AMT-130 in up to 6 patients with lower striatal volumes compared to those of patients enrolled in previous cohorts. Additional details are available on www​.clin​i​cal​tri​als​.gov (NCT04120493).

GenTLE is a Phase I/IIa multi-center, open-label trial being conducted in the U.S. to evaluate the safety, tolerability and exploratory signs of efficacy of two doses of AMT-260 in individuals with refractory MTLE. The study comprises two dose cohorts of six patients each. The study is actively recruiting through 17 sites. In November 2024, we announced that the first patient had been dosed in the trial.

The Phase I/II trial of AMT-191 is an open-label multi-center study to evaluate safety and biomarkers of efficacy of a single dose of intravenously-administered AMT-191. The study will also include exploratory functional efficacy assessments. The plan is to investigate 2 sequential dose cohorts in 3–6 Participants per cohort. Participants will be monitored for 24 hours following AMT-191 administration then follow-up study visits will continue for 24 months, during which safety, pharmacokinetics/pharmacodynamics, biomarkers, and efficacy assessments will be performed. Participants will continue receiving their regularly scheduled enzyme replacement therapy (ERT) until they meet the criteria for withdrawal.

The Phase I/II trial of AMT-162 in participants with SOD1-ALS is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. It is a Phase 1/2, multi-center, single ascending dose study.

AMT-162 is an investigational gene therapy that encodes an artificial microribonucleic acid (microRNA or miRNA) targeting the SOD1 gene. This clinical study will test the safety of AMT-162 and explore the hypothesis that it will silence expression of mutant cytosolic SOD1 and thereby ameliorate the course of ALS caused by this mutant gene.