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

Running, not enrolling · Phase 2 · Has a placebo group

Conditions studied: Huntington Disease

In brief

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).

Key facts

Study ID

NCT05686551

Run by

Hoffmann-La Roche

People needed

301

Starts

2023-02-03

Expected to finish

2027-04-01

Last updated by the study team

2026-05-22

Who can join

Age: 25 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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
• Current or previous use of an ASO other than tominersen (including small interfering RNA) or any other HTT-lowering therapy
• Hydrocephalus
• Received any active investigational treatment other than tominersen during or since completion of the DB treatment period
• Key inclusions/exclusion criteria are listed here. Other protocol-defined I/E criteria may apply.

Where it is running

• 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
• Beth Israel Deaconess Medical Center — Boston, Massachusetts, United States
• Henry Ford Hospital — Detroit, Michigan, United States
• Dent Neurological Institute — Amherst, New York, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• The University of Texas Health Science Center at Houston — Houston, Texas, United States
• EvergreenHealth Investigational Drug Services — Kirkland, Washington, United States
• Inland Northwest Research — Spokane, Washington, United States
• CINME — Buenos Aires, Argentina
• Hospital Ramos Mejía — CABA, Argentina
• INEBA — Capital Federal, Argentina
• Hospital Britanico de Buenos Aires — Ciudad Autonoma Buenos Aires, Argentina
• Monash Medical Centre — Clayton, Victoria, Australia
• Royal Melbourne Hospital — Parkville, Victoria, Australia
• Perron Institute for Neurological and Translational Science — Nedlands, Western Australia, Australia
• Uab Medicine — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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