Safety, pharmacokinetics and pharmacodynamics of SBT-020 in patients with early stage Huntington's disease, a 2-part study.
Marcus PJ van Diemen, Ellen P Hart, Anthony Abbruscato, Liz Mead, Ilse van Beelen, Sandrin C Bergheanu, Pieter W Hameeteman, Emma Coppen, Jessica Y Winder, Matthijs Moerland, Hermien Kan, Jeroen van der Grond, Andrew Webb, Raymund AC Roos, Geert Jan Groeneveld
Br J Clin Pharmacol | Published : 2021
AIMS: Huntington's disease (HD) is a neurodegenerative disease with cognitive, motor and psychiatric symptoms. Toxic accumulation of misfolded mutant huntingtin protein induces mitochondrial dysfunction, leading to a bioenergetic insufficiency in neuronal and muscle cells. We evaluated the safety, pharmacokinetics and pharmacodynamics of SBT-020, a novel compound to improve mitochondrial function, in a 2-part study in early stage HD patients. METHODS: Part 1 consisted of 7-day multiple ascending dose study to select the highest tolerable dose for Part 2, a 28-day multiple dose study. Mitochondrial function was measured in the visual cortex and calf muscle, using phosphorous magnetic resonanc..View full abstract