This was a Phase I/II clinical study, which means it was the first study to test this treatment in people. In clinical research, at this stage, the main purpose of the study is to evaluate if the treatment is safe and to look for early signs that it might be beneficial.
The study followed participants for three years (36 months) and included people with early-stage HD who received either a low or high dose of AMT-130. The experimental treatment was delivered through brain surgery, performed by an experienced neurosurgical team. The published results focus on 12 participants from the high-dose group who have completed the full 36-month follow-up.
Because HD is rare and the administration of the treatments involves brain surgery, this study did not include a placebo group. Instead, researchers used what is known as an external control group, meaning they compared the results of people who received AMT-130 with previously collected data from other individuals with HD who are part of the Enroll-HD database. In studies that use an external control group, participants are matched with people of similar age, symptoms, and disease stage to make the comparison as fair as possible.
To assess how well the treatment might be working researchers looked at standard clinical measures commonly used in HD studies, which evaluate motor, cognitive, and functional abilities. It’s important to note that these results are based on average scores across the group, not on individual outcomes. Because this is a small group of only 12 participants, each person’s data has a big impact on the overall results, meaning some people may have improved more than others.
