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Purpose

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease (HD).

Condition

Eligibility

Eligible Ages
Between 25 Years and 50 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive 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

  • Current or previous use of an antisense oligonucleotide (ASO) (including small interfering ribonucleic acid [RNA]) or any huntingtin gene/protein (HTT) lowering therapy (including tominersen) - 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Tominersen 60 milligrams (mg)
  • Drug: Tominersen 60 mg
    60 mg tominersen administered intrathecally every 16 weeks (Q16W).
    Other names:
    • RO7234292;
    • RG6042
Placebo Comparator
Placebo
  • Drug: Placebo
    Matching placebo administered intrathecally Q16W.
Experimental
Tominersen 100 mg
  • Drug: Tominersen 100 mg
    100 mg tominersen administered intrathecally Q16W.
    Other names:
    • RO7234292;
    • RG6042

More Details

Status
Active, not recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.