BN40955 OLE study to evaluate the safety & tolerability of RO7234292
Research type
Research Study
Full title
AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF INTRATHECALLY ADMINISTERED RO7234292 (RG6042) IN PATIENTS WITH HUNTINGTON’S DISEASE
IRAS ID
257158
Contact name
Sarah Tabrizi
Contact email
Sponsor organisation
F Hoffmann-La Roche Ltd
Eudract number
2018-003898-94
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
DEME 40655, NIHR CRN Reference Number
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
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.To date, there are no approved treatments able to slow or stop the clinical progression of HD. Currently approved treatments aim to reduce the burden of symptoms, maximize function, and improve quality of life.
RO7234292 (study drug) is designed to target the cause of HD and offers the potential to meet this unmet medical need. This study is designed to assess the long-term safety and tolerability of RO7234292 given intrathecally (into the spine by a lumbar puncture) in patients who have completed other F. Hoffmann-La Roche, Ltd.sponsored (hereafter referred to as Roche) and/or Genentech-sponsored studies in HD in the RO7234292 development program.
Current planned or ongoing studies are designed for durations of up to approximately 2 years. This study is designed to collect safety data in a larger number of patients and will also study the benefit and potential risks of RO7234292 when administered every 4 or 8 weeks over a longer period of time.
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 treatment will return to the clinic approximately 4 weeks after their last dose of study drug.
In the UK it is expected that approximately 21 participants will be enrolled.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/0109
Date of REC Opinion
7 Mar 2019
REC opinion
Further Information Favourable Opinion