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
Condition: Huntington Disease
MeSH terms: Huntington Disease
Intervention: RO7234292 (RG6042)
Intervention description: Intrathecal injection
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 236.0
Sex: All
Minimum age: 25 Years
Healthy volunteers: No

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.
    

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

        Inclusion criteria of 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

        Exclusion criteria of 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 relation to the COVID-19 pandemic:

          -  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