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.
- Conditions
- Huntington Disease
- Interventions
- Registration Number
- NCT05686551
- Lead Sponsor
- Hoffmann-La Roche
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 300
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo - Tominersen 60 mg Tominersen 60 mg - Tominersen 100 mg Tominersen 100 mg -
- Primary Outcome Measures
Name Time Method Change from baseline in clinical laboratory results - Cerebrospinal fluid (CSF) White Blood Cell (WBC) (1/uL) From Baseline Visit (Day 1), and Months 4, 8, 9, 12, 16 Change in baseline in structural MRI assessing any new abnormalities including radiographic features consistent with hydrocephalus and other relevant MRI safety findings From Baseline, Months 4, 8, 12, 16 and Up to Approximately Month 24 Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale Up to Approximately 24 Months Change from baseline in clinical laboratory results Cerebrospinal fluid (CSF) protein (g/L) From Baseline Visit (Day 1), and Months 4, 8, 9, 12, 16 Percentage change from baseline in geometric means of CSF mHTT protein levels at Month 9 Baseline and Month 9 Change from baseline in composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. sites) at 16 months Baseline to 16 Months Change in scores on the scale
Change from baseline in Total Functional Capacity (TFC) Scores (U.S. sites) at 16 months Baseline to 16 Months Change in scores on the scale
- Secondary Outcome Measures
Name Time Method Change from baseline in MoCA Scores From Baseline, Months 4, 8, 12, 16 and up to approximately Month 24 Percentage of participants with suicidal ideation or behavior as assessed by C-SSRS score at each visit, including detailed focus on any individual cases identified as having severe ideation or behavior during the study conduct Up to Approximately 24 Months Change from baseline at 16 months for the assessments of TFC (non-U.S. sites) Scores Baseline to 16 Months Change from baseline at 16 months for the assessments of cUHDRS (U.S. sites) Scores Baseline to 16 Months Change from baseline at 16 months for the assessments of Symbol Digit Modalities Test (SDMT) Scores Baseline to 16 Months Change from Baseline at 16 Months for the Assessments of Stroop Word Reading (SWR) Scores Baseline to 16 Months Change from baseline at 16 months for the assessments of Total Motor Score (TMS) Baseline to 16 Months Change from baseline in CSF Neurofilament light Chain (NfL) levels at 16 months Baseline to 16 Months Incidence of anti-drug antibodies (ADAs) at specified timepoints relative to the prevalence of ADAs at baseline From Baseline, Months 4, 8, 12, 16 and Up to Approximately Month 24 Titers determined if ADAs are identified From Baseline, Months 4, 8, 12, 16 and Up to Approximately Month 24
Trial Locations
- Locations (73)
Uab Medicine
🇺🇸Birmingham, Alabama, United States
Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
University of California San Diego
🇺🇸La Jolla, California, 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, 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
Westmead Hospital
🇦🇺Westmead, New South Wales, Australia
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 Cedex 9, France
Groupe Hospitalier Pellegrin
🇫🇷Bordeaux, France
Hopital Henri Mondor
🇫🇷Creteil, 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 Hôpital 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
🇮🇹Roma, Lazio, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
🇮🇹Milano, Lombardia, Italy
New Zealand Brain Research Institute
🇳🇿Christchurch, New Zealand
Waikato Hospital
🇳🇿Hamilton, New Zealand
Szpital Sw. Wojciecha
🇵🇱Gda?sk, Poland
Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii K
🇵🇱Krakow, Poland
Wojskowy Instytut Medycyny Lotniczej
🇵🇱Warszawa, Poland
Instytut Psychiatrii i Neurologii
🇵🇱Warszawa, Poland
Hospital de Santa Maria
🇵🇹Lisboa, 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
🇪🇸Sevilla, 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