BN40423 Generation HD 1
Research type
Research Study
Full title
A RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE III CLINICAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INTRATHECALLY ADMINISTERED RO7234292 (RG6042) IN PATIENTS WITH MANIFEST HUNTINGTON'S DISEASE
IRAS ID
251414
Contact name
Sarah Tabrizi
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2018-002987-14
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
4 years, 8 months, 1 days
Research summary
Summary of Research
Huntington's disease (HD) is a neurodegenerative disease causing progressive motor, cognitive, and functional decline usually in adult life, with an average age of motor onset of 45 years. The average illness course after motor onset is approximately 10-20 years, with pneumonia, heart failure, or other complications frequently cited as the cause of death. Individuals with end-stage disease have complete physical disability and profound body wasting.
The prevalence of Huntington's disease in North America, northwestern Europe, and Australia ranges from an estimated 5.96 to 13.17 cases per 100,000.This is a Phase III, randomized, placebo-controlled, double-blind, multicenter clinical study to evaluate the efficacy and safety of a new drug - RO7234292 in patients with manifest HD.
Approximately 660 patients will be enrolled in the study globally. Prospective patients will undergo screening assessments during a 4-week screening period and patients that are eligible will be randomly allocated to receive RO7234292 every 4 weeks, placebo every 4 weeks or RO7234292/placebo alternating every 4 weeks by Intrathecal injection.
Patients will undergo evaluations during the study that include neurologic examinations, vital signs, ECGs, clinical laboratory tests, questionnaires, neuroimaging assessments (neurologic safety sequences), and adverse events including related concomitant medications. Patients who complete the treatment period will return to the clinic for an end-of-treatment visit at Week 101 (approx 2 years).
Patients will then be given the option on an individual basis of receiving RO7234292 in an Open Label Extension study (BN40955) upon completion of Study BN40423, provided they meet eligibility criteria and the data from the ongoing RO7234292 program support continued development.
The study is expected to last for approximately 4 years. In the UK it is expected that up to 50 participants will be enrolled.
Summary of Results
The lay summary of study results will be available from 24th March 2023 on clinicaltrials.gov:
https://clinicaltrials.gov/ct2/show/results/NCT03761849?term=NCT03761849&draw=2&rank=1&view=results
and the Roche for patients platform:
https://forpatients.roche.com/en/trials/neurodegenerative-disorder/hd/a-study-to-evaluate-the-efficacy-and-safety-of-intrathe-26435.html
REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/2136
Date of REC Opinion
7 Feb 2019
REC opinion
Further Information Favourable Opinion