NCT05686551

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

A Phase II, Randomized, Double-blind, Placebo-controlled, Dose-finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington's Disease

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 301 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 25 Years – 50 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).

Conditions

• Huntington Disease

Interventions

Type	Name	Description
DRUG	Tominersen	Tominersen will be administered at the dose and schedule specified in the protocol.
DRUG	Placebo	Matching placebo administered IT, Q16W during the DB period.

Timeline

• Start date: 2023-02-03
• Primary completion: 2026-05-31
• Completion: 2027-04-01
• First posted: 2023-01-17
• Last updated: 2026-03-02

Locations

70 sites across 15 countries: United States, Argentina, Australia, Austria, Canada, Denmark, France, Germany, Italy, New Zealand, Poland, Portugal, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study