An open-label study to assess the feasibility and tolerability of rilmenidine for the treatment of Huntington's disease

Journal of Neurology
Benjamin R UnderwoodRoger A Barker

Abstract

Preclinical data have shown that rilmenidine can regulate autophagy in models of Huntington's disease (HD), providing a potential route to alter the disease course in patients. Consequently, a 2-year open-label study examining the tolerability and feasibility of rilmenidine in mild-moderate HD was undertaken. 18 non-demented patients with mild to moderate HD took daily doses of 1 mg Rilmenidine for 6 months and 2 mg for a further 18 months followed by a 3-month washout period. The primary outcome was the number of withdrawals and serious adverse events. Secondary outcomes included safety parameters and changes in disease-specific variables, such as motor, cognitive and functional performance, structural MRI and serum metabolomic analysis. 12 patients completed the study; reasons for withdrawal included problems tolerating study procedures (MRI, and venepuncture), depression requiring hospital admission and logistical reasons. Three serious adverse events were recorded, including hospitalisation for depression, but none were thought to be drug-related. Changes in secondary outcomes were analysed as the annual rate of change in the study group. The overall change was comparable to changes seen in recent large observational studie...Continue Reading

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Citations

Mar 16, 2018·Metabolic Brain Disease·Karolina PierzynowskaGrzegorz Węgrzyn
Apr 30, 2019·Frontiers in Neuroscience·Yanfeng JiangXingdong Chen
Mar 10, 2020·Journal of Neurochemistry·Olga CortiPhilip M Beart
Jan 15, 2019·Journal of Huntington's Disease·Melanie P Jensen, Roger A Barker
Feb 8, 2019·Cell Death and Differentiation·Maria Chiara Maiuri, Guido Kroemer
Jul 10, 2021·Autophagy·Muhammed KocakOle Pless
Aug 28, 2021·International Journal of Molecular Sciences·Amy KimJoana Gil-Mohapel

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Software Mentioned

COHORT
TRACK
HD
R
CANTAB
Stan

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