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
NCT ID: NCT05686551
Last Updated: 2025-11-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
301 participants
INTERVENTIONAL
2023-02-03
2027-04-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Tominersen 60 milligrams (mg)
Tominersen 60 mg
60 mg tominersen administered intrathecally every 16 weeks (Q16W).
Placebo
Placebo
Matching placebo administered intrathecally Q16W.
Tominersen 100 mg
Tominersen 100 mg
100 mg tominersen administered intrathecally Q16W.
Interventions
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Tominersen 60 mg
60 mg tominersen administered intrathecally every 16 weeks (Q16W).
Placebo
Matching placebo administered intrathecally Q16W.
Tominersen 100 mg
100 mg tominersen administered intrathecally Q16W.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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/m2)
* Study companion
Exclusion Criteria
* 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
25 Years
50 Years
ALL
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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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
Countries
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References
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McColgan P, Thobhani A, Boak L, Schobel SA, Nicotra A, Palermo G, Trundell D, Zhou J, Schlegel V, Sanwald Ducray P, Hawellek DJ, Dorn J, Simillion C, Lindemann M, Wheelock V, Durr A, Anderson KE, Long JD, Wild EJ, Landwehrmeyer GB, Leavitt BR, Tabrizi SJ, Doody R; GENERATION HD1 Investigators. Tominersen in Adults with Manifest Huntington's Disease. N Engl J Med. 2023 Dec 7;389(23):2203-2205. doi: 10.1056/NEJMc2300400. No abstract available.
Other Identifiers
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Other
Identifier Type: OTHER
Identifier Source: secondary_id
2022-001991-32
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-503928-10-00
Identifier Type: CTIS
Identifier Source: secondary_id
BN42489
Identifier Type: -
Identifier Source: org_study_id
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