Clinical Trials

In the multi-center, Phase I/II clinical trial of AMT-130 currently being conducted in the U.S., a total of 26 patients with early-manifest Huntington’s disease have been enrolled, including an initial 10-patient low-dose cohort (6 treated, 4 control) with up to 30 months of follow-up and a subsequent 16-patient high-dose cohort (10 treated, 6 control) with up to 18 months of follow-up. 

Patients were randomized to treatment with AMT-130 or an imitation (sham) surgery. The U.S. study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of five years for treated patients. Four of the six control patients in the high-dose cohort were crossed over to treatment and the remaining two patients failed to meet the study’s inclusion criteria. (ClinicalTrials​.gov Identifier: NCT04120493).

The multi-center, open-label, Phase I/II clinical trial of AMT-130 being conducted in Europe and the UK enrolled a total of 13 patients with the same early manifest criteria for Huntington’s disease as the U.S. study. Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the subsequent high-dose cohort. 

The first-in-human Phase I/IIa clinical trial will be conducted in the United States and consist of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept (POC) data. Patient enrollment in the trial is expected to begin in third quarter of 2024.

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.