GENERATION HD2

GENERATION HD2 is active, but no longer recruitingA Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared with Placebo in Participants with Prodromal and Early Manifest Huntington’s Disease

Find this study on clinicaltrials.gov and the Roche website.

SponsorHoffmann-La Roche
The StudyTo evaluate the safety, biomarkers, and efficacy of a new drug, tominersen, in participants with prodromal (early signs) or early manifest (mild symptoms) of Huntington's Disease, focusing on individuals ages 25 to 50. The goal is to compare different doses of tominersen with a placebo to slow down disease progression.

Tominersen is an investigational drug that works by targeting the genetic material (RNA) that makes huntingtin protein, to lower production of both the normal and mutant (expanded CAG) forms. The study builds on the previous GENERATION HD1 trial, which tested higher doses of tominersen in people with more advanced HD. While that trial didn’t meet its main goals, it suggested that lower doses may be more helpful for younger individuals in the earlier stages of HD.
PhaseI, dose-finding