Study of ALN-HTT02 Given Into the Spine in Adults Aged 25 to 70 With Huntingtons Disease to Test Safety and How the Drug Works

What this Trial Is About

Researchers are evaluating the safety, tolerability, pharmacodynamics, and pharmacokinetics of ALN-HTT02 in adult patients with Huntingtons Disease. This phase 1 study focuses on patients with stage 2 or early stage 3 Huntingtons Disease. Participants are randomly assigned to receive either ALN-HTT02 or a placebo in a double-blind manner to assess the effects of this intrathecally administered drug. Participants receive a single dose of ALN-HTT02 or placebo during the double-blind part of the study. Those who received ALN-HTT02 initially will not get another dose in the open-label part, while placebo recipients may receive a single ALN-HTT02 dose then. Additionally, participants can opt for repeat doses of ALN-HTT02 in an open-label extension lasting up to 36 months. Throughout the study, participants undergo monitoring for adverse events for up to 12 months during the double-blind and open-label parts, and up to 36 months in the extension. Researchers measure levels of mutant huntingtin protein in cerebrospinal fluid, as well as concentrations of ALN-HTT02 in plasma, cerebrospinal fluid, and urine. Safety, pharmacologic effects, and long-term tolerability are carefully assessed during this extended participation period.

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease