Summary

Eligibility
for people ages 25-50 (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
completion around

Description

Summary

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

Official Title

A Phase II, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington's Disease

Keywords

Huntington Disease, Prodromal and Early Manifest Huntington's Disease, Tominersen 60 mg, Tominersen 100 mg

Eligibility

You can join if…

Open to people ages 25-50

-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

You CAN'T join if...

  • 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

Locations

  • University of California San Diego in progress, not accepting new patients
    La Jolla California 92037-1337 United States
  • Barrow Neurological Institute accepting new patients
    Phoenix Arizona 85013 United States
  • University of California Davis Medical System accepting new patients
    Sacramento California 95817 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Hoffmann-La Roche
ID
NCT05686551
Phase
Phase 2 Huntington's Disease Research Study
Study Type
Interventional
Participants
Expecting 300 study participants
Last Updated