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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.

300 patients around the world

Available in Argentina, United States, Spain

Hoffmann-La Roche

9Research sites

300Patients around the world

This study is for people with

Rare diseases

Huntington's chorea

Requirements for the patient

To 50 Years

All Gender

Medical requirements

: Huntington's disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive
Prodromal HD (defined as DCL 2 to 3, Independence Scale (IS) ⩾70, and ⩾TFC8)
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

: 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

Sites

Centro de Investigaciones Metabólicas (CINME) S.A. - CABA, Buenos Aires

Recruiting

Viamonte, Cdad. Autónoma de Buenos Aires, Argentina

INEBA

Recruiting

Guardia Vieja 4435, C1192AAW CABA, Argentina

Hospital Universitario de Badajoz - HUB

Recruiting

Av. de Elvas, s/n, 06080 Badajoz

Hospital Santa Creu i Sant Pau

Recruiting

C/ de Sant Quintí, 77, 08041 Barcelona, Spain

Hospital Universitario de Burgos

Recruiting

Av. Islas Baleares, 3, 09006 Burgos

Hospital Universitario Ramón y Cajal

Recruiting

Madrid, 28034

Hospital Universitario Virgen de la Macarena

Recruiting

Avenida Dr. Fedriani, 3, 41009, Sevilla

Hospital Universitari i Politecnic La Fe

Recruiting

46026 Valencia, Spain

Hospital Universitario de Cruces

Recruiting

Cruces Plaza, s/n, 48903 Barakaldo, Bizkaia

SponsorHoffmann-La Roche

Study typeInterventional

Conditions

Huntington's chorea

Requirements

To 50 YearsAll Gender

Unique study IDclinicaltrials.gov

NCT05686551