An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies

NCT ID: NCT03842969

Last Updated: 2023-08-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 236 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-04-23
• Study Completion Date: 2022-03-30

Brief Summary

This study will evaluate the long-term safety and tolerability of RO7234292 (RG6042) in participants who have completed other F. Hoffmann-La Roche, Ltd.-sponsored and/or Genentech-sponsored studies in the Huntington's disease (HD) in the development program for RG6042.

Detailed Description

Entry into the study should occur at the time the participant completes participation in one of the preceding studies. Upon completion of the inclusion visit, eligible participants will receive either RO7234292 (RG6042) every 8 weeks (Q8W) or RO7234292 (RG6042) every 16 weeks (Q16W) by the bolus intrathecal injection.

Conditions

Huntington Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

RO7234292 (RG6042) Q8W

Participants who received open-label RO7234292 Q4W in a preceding study or who received RO7234292 Q4W in this study may be randomly allocated to receive RO7234292 Q8W. Participants who previously received open-label of RO7234292 Q8W in a preceding study or are currently receiving RO7234292 Q8W in this study will receive RO7234292 Q8W. Participants who previously received placebo, or did not previously receive treatment with RO7234292 or received short-term treatment with a treatment-free follow-up period may be randomly allocated to receive RO7234292 Q8W. Participants who previously received blinded placebo Q8W may receive RO7234292 Q8W. Participants who received blinded RO7234292 Q8W will receive open-label RO7234929 Q8W. Participants who received blinded placebo Q8W may receive RO7234292 Q8W. Participants who received blinded RO7234292 Q4W or blinded placebo Q4W may be randomly allocated to receive open-label of RO7234292 Q8W.

Group Type: EXPERIMENTAL

RO7234292 (RG6042)

Intervention Type: DRUG

Intrathecal injection

RO7234292 (RG6042) Q16W

Participants who previously received open-label RO7234292 Q4W in a preceding study or who received RO7234292 Q4W in this study may be randomly allocated to receive RO7234292 Q16W. Participants who previously received placebo in a preceding study or did not previously receive treatment with RO7234292 or received short-term treatment with a treatment-free follow-up period may be randomly allocated to receive RO7234292 Q16W. Participants who previously received blinded placebo Q8W will receive RO7234292 Q8W. Participants who previously received blinded RO7234292 Q16W will receive open-label RO7234292 Q16W. Participants who received blinded RO7234292 Q4W or blinded placebo Q4W may be randomly allocated to receive open-label of RO7234292 Q16W. Participants who previously received open-label RO7234292 Q16W will receive open-label RO7234292 Q16W.

Group Type: EXPERIMENTAL

RO7234292 (RG6042)

Intervention Type: DRUG

Intrathecal injection

Interventions

RO7234292 (RG6042)

Intrathecal injection

Intervention Type: DRUG

Other Intervention Names

Tominersen

Eligibility Criteria

Inclusion Criteria

• Prior enrollment in a Roche-sponsored or Genentech-sponsored study in HD for the RO7234292 (RG6042) development program that made provision for entry into an OLE study
• For women of childbearing potential: agreement to remain abstinent or use contraceptive measures
• For men: agreement to remain abstinent or use a condom, and agreement to refrain from donating sperm
• Patients who were screened and eligible for the placebo-controlled Phase III Study BN40423 but could not be randomized prior to the close of Study BN40423 enrollment due to challenges relating to the COVID-19 pandemic

• 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

• Withdrawal of consent from the preceding study
• Permanent discontinuation of RO7234292 (RG6042) for any drug-related safety concern during the preceding study or meeting of any study treatment discontinuation criteria specified in the preceding study at the time of enrollment into this study
• An ongoing, unresolved, clinically significant medical problem that in the judgment of the investigator would make it unsafe for the patient to participate in this study
• Antiplatelet or anticoagulant therapy within 14 days prior to inclusion or anticipated use during the study, including, but not limited to, aspirin, clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban
• History of bleeding diathesis or coagulopathy
• Platelet count less than the lower limit of normal
• Concurrent participation in any therapeutic clinical trial
• Study treatment (RO7234292/RG6042) is commercially marketed in the patient's country for the patient-specific disease and is accessible to the patient
• Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug

• 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
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hoffmann-La Roche

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

Uab Medicine

Birmingham, Alabama, United States

University of California San Diego

La Jolla, California, United States

Stanford Univ Medical Center

Palo Alto, California, United States

SC3 Research Group, Inc

Pasadena, California, United States

CenExel Rocky Mountain Clinical Research, LLC

Englewood, Colorado, United States

Georgetown University; Research Division, Psychiatry

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

University of South Florida

Tampa, Florida, United States

Northwestern University

Chicago, Illinois, United States

John Hopkins University School of Medicine

Baltimore, Maryland, United States

Dent Neurological Institute

Amherst, New York, United States

Columbia University

New York, 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; McGovern Medical School

Houston, Texas, United States

Uniklinik fuer Neurologie, Medizinische Universitaet Innsbruck; Department fuer Neurologie

Innsbruck, , Austria

University of British Columbia Hospital; Division of Neurology

Vancouver, British Columbia, Canada

Centre for Movement Disorders

North YORK, Ontario, Canada

Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

Centre Hospitalier de l?Université de Montréal (CHUM)

Montreal, Quebec, Canada

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

Berlin, , Germany

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

Bochum, , Germany

Universitätsklinikum Ulm; Klinik für Neurologie

Ulm, , Germany

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

San Giovanni Rotondo (FG), Apulia, Italy

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

Milan, Lombardy, Italy

Universitair Medisch Centrum Groningen

Groningen, , Netherlands

LUMC

Leiden, , Netherlands

Hospital Universitario de Badajoz; Servicio de Neurología

Badajoz, , Spain

Hospital Clinic Servicio de Neurologia

Barcelona, , Spain

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

Barcelona, , Spain

Hospital Universitario de Burgos. Servicio de Neurología

Burgos, , Spain

Hospital Ramon y Cajal; Servicio de Neurologia

Madrid, , Spain

Fundacion Jimenez Diaz; Servicio de Neurología

Madrid, , Spain

Hospital Universitario Virgen Macarena; Servicio de Neurologia

Seville, , Spain

Birmingham and Solihull Mental Health Foundation Trust; Institute of Clinical Sciences

Birmingham, , United Kingdom

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

Cambridge, , United Kingdom

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

Cardiff, , United Kingdom

University College London Hospitals NHS Foundation Trust - University College Hospital

London, , United Kingdom

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

Manchester, , United Kingdom

Countries

United States; Austria; Canada; Germany; Italy; Netherlands; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2018-003898-94

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

BN40955

Identifier Type: -

Identifier Source: org_study_id