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ACTIVE NOT RECRUITING

NCT05686551

PHASE2

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

Sponsor: 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).

Official title: 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

Key Details

Gender

All

Age Range

25 Years - 50 Years

Study Type

INTERVENTIONAL

Enrollment

301

Start Date

2023-02-03

Completion Date

2027-04-01

Last Updated

2026-03-02

Healthy Volunteers

Conditions

Huntington Disease

Interventions

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.

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 * 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.

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

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

Uniklinik fuer Neurologie, Medizinische Universitaet Innsbruck

Innsbruck, Austria

University of Alberta Hospital

Edmonton, Alberta, Canada

Montreal Neurological Inst

Montreal, Quebec, Canada

Rigshospitalet, Hukommelsesklinikken

København Ø, Denmark

CHU Angers, Batiement Larrey 2, Neurologie

Angers, France

Groupe Hospitalier Pellegrin

Bordeaux, France

Hopital Henri Mondor

Créteil, France

Hopital Roger Salengro Service de Neurologie

Lille, France

CHU de la Timone - Hopital d Adultes

Marseille, France

Hopital Gui de Chauliac

Montpellier, France

CHU Strasbourg Hpital Hautepierre

Strasbourg, France

CHU toulouse - Hôpital Purpan

Toulouse, France

Uniklinik RWTH Aachen

Aachen, Germany

Charité - Universitätsmed. Berlin, Klinik für Psychiatrie und Psychotherapie

Berlin, Germany

St. Josef-Hospital, Neurologische Klinik der Ruhr-Uni

Bochum, Germany

German Center for Neurodegenerative Diseases (DZNE)

Bonn, Germany

Universitätsklinikum Erlangen, Abteilung Molekulare Neurologie

Erlangen, Germany

Universitätsklinikum Schleswig-Holstein / Campus Lübeck

Lübeck, Germany

kbo - Isar-Amper-Klinikum Taufkirchen

Taufkirchen, Germany

Universitätsklinikum Ulm

Ulm, Germany

Ospedale Bellaria

Bologna, Emilia-Romagna, Italy

Azienda Ospedaliera Sant'Andrea

Rome, Lazio, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Lombardy, Italy

New Zealand Brain Research Institute

Christchurch, New Zealand

Waikato Hospital

Hamilton, New Zealand

Szpital Sw. Wojciecha

Gdansk, Poland

Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K

Krakow, Poland

Wojskowy Instytut Medycyny Lotniczej

Warsaw, Poland

Hospital de Santa Maria

Lisbon, Portugal

CNS - Campus Neurológico

Torres Vedras, Portugal

Hospital de Cruces

Barakaldo, Vizcaya, Spain

Hospital Universitario de Badajoz

Badajoz, Spain

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

Hospital Universitario de Burgos. Servicio de Neurología

Burgos, Spain

Hospital Ramon y Cajal

Madrid, Spain

Hospital Universitario Virgen Macarena

Seville, Spain

Hospital Universitario la Fe

Valencia, Spain

Universitätsspital Basel

Basel, Switzerland

Neurozentrum Siloah

Gümligen, Switzerland

University Hospitals Birmingham NHS Foundation Trust

Birmingham, United Kingdom

Addenbrookes Hospital

Cambridge, United Kingdom

Chapel Allerton Hospital

Leeds, United Kingdom

UCL Hospital NHS Trust

London, United Kingdom

John Radcliffe Hospital

Oxford, United Kingdom

Southampton University Hospitals NHS Trust

Southampton, United Kingdom