BN42489 - TOMINERSEN IN PATIENTS WITH HD-ISS STAGE 2 & 3 HUNTINGTON’S DISEASE
Research type
Research Study
Full 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
IRAS ID
1006687
Contact name
Ethan Montenegro
Contact email
Sponsor organisation
F.Hoffmann-La Roche Ltd
Eudract number
2022-001991-32
Clinicaltrials.gov Identifier
Research summary
Presently, there are no therapies available to stop or slow the clinical progression of Huntington’s disease (HD), which is relentless until patients experience premature death. This study is testing the drug tominersen, which suppresses production of normal and mutant huntingtin protein, therefore aiming to prevent the downstream effects of the toxic mutant huntingtin protein and stop or slow the clinical progression of HD.\n\nThis study aims to evaluate the safety, biomarkers, and efficacy of three treatment groups which participants will be randomly allocated into: 60mg tominersen, 100mg tominersen, and placebo. The study aims to recruit 360 male and female participants globally, aged 25-50 years with early HD (pre-motor and early manifest). There will be approximately 18 patients recruited at 6 UK sites.\n\nParticipants will receive their allocated treatment for at least 16 months, and then will continue on treatment until the last enrolled participant has reached 16 months of treatment and completed the study. \n\nDepending on the results of the study, after completion of the treatment period for all participants an optional extension period may follow during which all participants receive the same dose of tominersen, selected based on results of the prior treatment period.\n\nThe study is sponsored by F. Hoffmann- La Roche.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
22/SW/0168
Date of REC Opinion
8 Feb 2023
REC opinion
Further Information Favourable Opinion