A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease

NCT ID: NCT03761849

Last Updated: 2024-03-01

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

899 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-01-23

Study Completion Date

2022-03-24

Brief Summary

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This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042) compared with placebo in participants with manifest Huntington's disease (HD)

Detailed Description

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Conditions

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Huntingtons Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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RO7234292 Q8W

RO4234292 is administered intrathecally every 8 weeks.

Group Type EXPERIMENTAL

RO7234292

Intervention Type DRUG

Intrathecal injection

RO7234292 Q16W

RO7234292 is administered intrathecally every 16 weeks. Participants in this arm will also receive placebo at alternate weeks to keep the blind.

Group Type EXPERIMENTAL

RO7234292

Intervention Type DRUG

Intrathecal injection

Placebo

Intervention Type DRUG

Intrathecal injection

Placebo

Placebo will be administered every 8 weeks by IT injection.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Intrathecal injection

Interventions

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RO7234292

Intrathecal injection

Intervention Type DRUG

Placebo

Intrathecal injection

Intervention Type DRUG

Other Intervention Names

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Tominersen

Eligibility Criteria

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Inclusion Criteria

* Manifest HD diagnosis, defined as a DCL score of 4
* Independence Scale (IS) score \>= 70
* Genetically confirmed disease by direct DNA testing with a CAP score \>400
* Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).

Exclusion Criteria

* Any serious medical condition or clinically significant laboratory, or vital sign abnormality or claustrophobia at screening that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
* Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
Minimum Eligible Age

25 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

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

Site Status

Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

University of California San Diego

La Jolla, California, United States

Site Status

Stanford Univ Medical Center

Palo Alto, California, United States

Site Status

SC3 Research Group, Inc

Pasadena, California, United States

Site Status

University of California Davis Medical System

Sacramento, California, United States

Site Status

CenExel Rocky Mountain Clinical Research, LLC

Englewood, Colorado, United States

Site Status

Georgetown University; Research Division, Psychiatry

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

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

John Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Dent Neurological Institute

Amherst, New York, United States

Site Status

Columbia University

New York, New York, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

The University of Texas Health Science Center at Houston; McGovern Medical School

Houston, Texas, United States

Site Status

University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, United States

Site Status

Evergreen Health Care Center

Kirkland, Washington, United States

Site Status

Hospital Ramos Mejía

CABA, , Argentina

Site Status

INEBA

Capital Federal, , Argentina

Site Status

Hospital Britanico de Buenos Aires

Ciudad Autonoma Buenos Aires, , Argentina

Site Status

WESTMEAD HOSPITAL; Deparment of Neurology

Westmead, New South Wales, Australia

Site Status

Monash Medical Centre

Clayton, Victoria, Australia

Site Status

Royal Melbourne Hospital; Department of Neurology

Parkville, Victoria, Australia

Site Status

Uniklinik fuer Neurologie, Medizinische Universitaet Innsbruck; Department fuer Neurologie

Innsbruck, , Austria

Site Status

Christian-Doppler-Klinik - Universitätsklinikum; Universitätskliniik für Neurologie

Salzburg, , Austria

Site Status

University of Alberta Hospital

Edmonton, Alberta, Canada

Site Status

University of British Columbia Hospital; Division of Neurology

Vancouver, British Columbia, Canada

Site Status

True North Clinical Research-Halifax

Halifax, Nova Scotia, Canada

Site Status

Centre for Movement Disorders

North YORK, Ontario, Canada

Site Status

Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

Site Status

Centre Hospitalier de l?Université de Montréal (CHUM)

Montreal, Quebec, Canada

Site Status

Centro de Trastornos del Movimiento (CETRAM); CETRAM

Santiago, , Chile

Site Status

Aarhus Universitetshospital; Neurologisk Afdeling F, Neurogenetisk Afsnit

Aarhus N, , Denmark

Site Status

Rigshospitalet, Hukommelsesklinikken

København Ø, , Denmark

Site Status

CHU Angers, Batiement Larrey 2, Neurologie

Angers, , France

Site Status

Hopital Henri Mondor; Service de Neurologie

Créteil, , France

Site Status

Hopital Roger Salengro Service de Neurologie

Lille, , France

Site Status

CHU de la Timone - Hopital d Adultes; Service de Neurologie

Marseille, , France

Site Status

Hopital Gui de Chauliac; Neurologie

Montpellier, , France

Site Status

Hôpital Pitié Salpêtrère; Département de Génétique et Cytogénétique

Paris, , France

Site Status

CHU toulouse - Hôpital Purpan; Departement de Neurologie

Toulouse, , France

Site Status

Uniklinik RWTH Aachen; Klinik für Neurologie

Aachen, , Germany

Site Status

Charité - Universitätsmed. Berlin, Klinik für Psychiatrie und Psychotherapie; Abt. Neuropsychiatrie

Berlin, , Germany

Site Status

St. Josef-Hospital, Neurologische Klinik der Ruhr-Uni; Huntington-Center NRW, Abt. Neurodegeneration

Bochum, , Germany

Site Status

German Center for Neurodegenerative Diseases (DZNE)

Bonn, , Germany

Site Status

Universitätsklinikum Erlangen, Abteilung Molekulare Neurologie

Erlangen, , Germany

Site Status

Universitätsklinikum Schleswig-Holstein / Campus Lübeck; Zentrum für Seltene Erkrankungen

Lübeck, , Germany

Site Status

George-Huntington- Institut GmbH; Technologiepark Münster

Münster, , Germany

Site Status

Universitätsklinikum Ulm; Klinik für Neurologie

Ulm, , Germany

Site Status

IRCCS Casa Sollievo Della Sofferenza; Unità Ricerca e Cura Huntington e Malattie Rare

San Giovanni Rotondo, Apulia, Italy

Site Status

IRCCS Istituto delle Scienze Neurologiche; UOC Clinica Neurologica

Bologna, Emilia-Romagna, Italy

Site Status

Azienda Ospedaliera Sant'Andrea; UOC Neurologia

Rome, Lazio, Italy

Site Status

Irccs A.O.U.San Martino Ist; Dinogmi

Genoa, Liguria, Italy

Site Status

Fondazione IRCCS Istituto Neurologico Carlo Besta; U.O.C. Genetica Medica-Neurogenetica

Milan, Lombardy, Italy

Site Status

A.O.U. Careggi; Diaprtimento Scienze Neurologiche e Psichiatriche

Florence, Tuscany, Italy

Site Status

Kuwana City Medical Center

Mie, , Japan

Site Status

National Hospital Organization Niigata National Hospital

Niigata, , Japan

Site Status

Okayama University Graduate School of Medicine, Densitry and Pharmaceutical Sciences.

Okayama, , Japan

Site Status

Osaka General Medical Center

Osaka, , Japan

Site Status

National Center of Neurology and Psychiatry

Tokyo, , Japan

Site Status

Universitair Medisch Centrum Groningen

Groningen, , Netherlands

Site Status

LUMC

Leiden, , Netherlands

Site Status

Auckland DHB - Neurlogy Department; Neurology Department

Auckland, , New Zealand

Site Status

New Zealand Brain Research Institute

Christchurch, , New Zealand

Site Status

Wellington Hospital; Department of Neurology

Wellington, , New Zealand

Site Status

Szpital Sw. Wojciecha; Oddzial Neurologiczny

Gdansk, , Poland

Site Status

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

Krakow, , Poland

Site Status

Instytut Psychiatrii i Neurologii

Warsaw, , Poland

Site Status

FSBHI Siberian Clinical Center of the Federal Medical and Biological Agency

Krasnoyarsk, Krasnoyarsk Krai, Russia

Site Status

Research Center of Neurology; Neurology Department #5

Moskva, Moscow Oblast, Russia

Site Status

?linical hospital at Kazan station, Republican Center for Movement Disorders and Botulinum Therapy

Kazan', Tatarstan Republic, Russia

Site Status

Hospital de Cruces; Servicio de Neurologia

Barakaldo, Vizcaya, Spain

Site Status

Hospital Universitario de Badajoz; Servicio de Neurología

Badajoz, , Spain

Site Status

Hospital Clinic Servicio de Neurologia

Barcelona, , Spain

Site Status

Hospital de la Santa Creu i Sant Pau; Servicio de Neurologia

Barcelona, , Spain

Site Status

Hospital Universitario de Burgos. Servicio de Neurología

Burgos, , Spain

Site Status

Hospital Ramon y Cajal; Servicio de Neurologia

Madrid, , Spain

Site Status

Fundacion Jimenez Diaz; Servicio de Neurología

Madrid, , Spain

Site Status

Hospital Universitario Virgen Macarena; Servicio de Neurologia

Seville, , Spain

Site Status

Hospital Universitario la Fe; Servicio de Neurologia

Valencia, , Spain

Site Status

Universitätsspital Basel; Neurologie

Basel, , Switzerland

Site Status

Neurozentrum Siloah

Gümligen, , Switzerland

Site Status

Aberdeen Royal Infirmary; Medical Genectics

Aberdeen, , United Kingdom

Site Status

Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

Cambridge Centre for Brain Repair; Department of Clinical Nuerosciences, Addenbrookes Hospital

Cambridge, , United Kingdom

Site Status

University Hospital of Wales; Division of Psychological Medicine and Clinical Neurosciences

Cardiff, , United Kingdom

Site Status

Queen Elizabeth University Hospital Glasgow

Glasgow, , United Kingdom

Site Status

Leeds General Infirmary

Leeds, , United Kingdom

Site Status

National Hospital For Neurology and Neurosurgery

London, , United Kingdom

Site Status

Central Manchester University Hospitals NHS Foundation Trust; Manchester Centre for Genomic Medicine

Manchester, , United Kingdom

Site Status

John Radcliffe Hospital

Oxford, , United Kingdom

Site Status

Royal Hallamshire Hospital

Sheffield, , United Kingdom

Site Status

University Hospital Southampton NHS Foundation Trust

Southhampton, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Canada Chile Denmark France Germany Italy Japan Netherlands New Zealand Poland Russia Spain Switzerland United Kingdom

References

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Rodrigues FB, Ferreira JJ, Wild EJ. Huntington's Disease Clinical Trials Corner: June 2019. J Huntingtons Dis. 2019;8(3):363-371. doi: 10.3233/JHD-199003.

Reference Type DERIVED
PMID: 31381524 (View on PubMed)

Rodrigues FB, Quinn L, Wild EJ. Huntington's Disease Clinical Trials Corner: January 2019. J Huntingtons Dis. 2019;8(1):115-125. doi: 10.3233/JHD-190001.

Reference Type DERIVED
PMID: 30776019 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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GENERATION HD1

Identifier Type: OTHER

Identifier Source: secondary_id

BN40423

Identifier Type: -

Identifier Source: org_study_id

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