GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

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About the study

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).
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Inclusion Criteria


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


Either:


  1. Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ⩾70, and TFC ⩾8); or
  2. Early manifest HD (defined as DCL 4, IS ⩾70, and TFC ⩾8);
  3. Total body weight > 40 kilograms (kg) and a body mass index (BMI) within the range of 18-32 kilograms per meter square (kg/m2)
  4. Study companion

EXCLUSION CRITERIA

Exclusion Criteria


  1. 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)
  2. 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
  3. History of gene therapy, cell transplantation, or brain surgery
  4. Hydrocephalus
  5. Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug
  6. 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

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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.

Study’s details


Contition
Huntington Disease
Age (in years)
25 - 50
Phase
PHASE2
Participants needed
301
Est. Completion Date
Apr 1, 2027
Treatment type
INTERVENTIONAL

Sponsor
Hoffmann-La Roche
ClinicalTrials.gov identifier
NCT05686551
Study number
BN42489

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