A Randomized, Double-blind, Placebo-controlled Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecal ALN-HTT02 in Adult Huntington's Disease Patients

What this Trial Is About

Researchers are evaluating ALN-HTT02, a drug given into the spinal fluid, in adults aged 25 to 70 years with stage 2 or early stage 3 Huntington's disease. The study aims to assess the safety, tolerability, how the drug moves through and affects the body (pharmacokinetics and pharmacodynamics), with a focus on single or repeat doses. This is a phase 1 randomized, double-blind, placebo-controlled trial sponsored by Alnylam Pharmaceuticals. Participants are randomly assigned to receive either a single dose of ALN-HTT02 or a placebo during the double-blind phase. Those who received the drug in this phase will not get another dose in the open-label phase, while those who received placebo can receive a single dose of ALN-HTT02 during the open-label phase. Afterward, all participants have the option to receive repeat doses in an open-label extension lasting up to 36 months. Throughout the study, participants will be monitored for adverse events for up to 12 months in each phase and up to 36 months during the extension. Researchers will measure mutant huntingtin protein levels in cerebrospinal fluid, and concentrations of ALN-HTT02 in plasma, cerebrospinal fluid, and urine at specified intervals. Safety, tolerability, and drug effects will be closely tracked over the course of the trial.

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