Loading clinical trials...

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 | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE2

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

NCT05686551•Hoffmann-La Roche

View on ClinicalTrials.gov

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).

Eligibility

Age

25 - 50 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•DB Period:
•HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive
•Either:
•* Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ≥70, and TFC ≥8); Or
•* Early manifest HD (defined as DCL 4, IS ≥70, and TFC ≥8)
•Total body weight \> 40 kilograms (kg) and a body mass index (BMI) within the range of 18-32 kilograms per meter square (kg/m\^2)
•Study companion
•OLE Period:
•Participants must have completed the DB treatment period
•Participants must remain in the DB Safety follow-up period until OLE period starts

Exclusion Criteria

•DB Period:
•Current or previous use of an antisense oligonucleotide (ASO) (including small interfering ribonucleic acid \[RNA\]) or any HTT lowering therapy (including tominersen)
•Anti-platelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study, including, but not limited to, aspirin (unless ≤ 81 milligrams per day \[mg/day\]), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban, and heparin
•History of gene therapy, cell transplantation, or brain surgery
•Hydrocephalus
•Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
•History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening
•OLE Period:
•Early discontinuation from the DB treatment and the safety follow-up (SFU) periods
•Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
+3 more criteria

Locations (70)

Uab Medicine

Birmingham, Alabama, United States

Barrow Neurological Institute

Phoenix, Arizona, United States

University of California Davis Medical System

Sacramento, California, United States

CenExel Rocky Mountain Clinical Research, LLC

Englewood, Colorado, United States

Georgetown University

Washington D.C., District of Columbia, United States

University of Florida

Gainesville, Florida, United States

University of South Florida

Tampa, Florida, United States

Northwestern University

Chicago, Illinois, United States

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

John Hopkins University School of Medicine

Baltimore, Maryland, United States

Key Dates

Start Date

February 3, 2023

Primary Completion Date

May 31, 2026

Completion Date

April 1, 2027

Last Updated

March 2, 2026

Enrollment

301

ACTUAL participants

Conditions

Huntington Disease

Interventions

Tominersen

DRUG

Placebo

DRUG

Study of BDNF Pathway Biomarkers in the Cerebrospinal Fluid in Patients With Huntington's Disease

NCT04012411

Huntington Disease

View study

RECRUITINGNA

Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington's Disease (MUSIC-HD)

NCT06414967

Huntington Disease

View study

RECRUITINGPHASE1/PHASE2

A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD

NCT05822908

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 2, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Study of BDNF Pathway Biomarkers in the Cerebrospinal Fluid in Patients With Huntington's Disease

Study to Evaluate Music Therapy on Irritability and Impulsivity in Patients With Huntington's Disease (MUSIC-HD)

A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD

IMarkHD: in Vivo Longitudinal Imaging of HD Pathology

Gene Therapy Development and Validation for Huntington's Disease Fibro TG-HD

An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)