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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.

Condition

Eligibility

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

Inclusion Criteria

  • Huntington's disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive Either: - Prodromal HD (defined as DCL 2 to 3, Independence Scale (IS) ⩾70, and ⩾TFC8); or - Early manifest HD (defined as DCL 4, Independence Scale (IS) ⩾70, and ⩾TFC8); - Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2 - Study Companion

Exclusion Criteria

  • Current or previous use of an ASO (including small interfering RNA) or any 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 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 mg
  • Drug: Tominersen 60 mg
    60 mg tominersen administered intrathecally every 16 weeks
Placebo Comparator
Placebo
  • Drug: Placebo
    Matching placebo administered intrathecally every 16 weeks
Experimental
Tominersen 100 mg
  • Drug: Tominersen 100 mg
    100 mg tominersen administered intrathecally every 16 weeks

Recruiting Locations

Uab Medicine
Birmingham, Alabama 35294

More Details

Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: BN42489 https://forpatients.roche.com/
888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com

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.