Study aims and goal: A Randomized, double-blind, placebo-controlled dose range finding study with Open-Label Extension to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of LMI070/branaplam in participants with early manifest HD.
No. of study sites: This Phase IIb study aims to recruit stage 1 and 2 participants at sites in Belgium, Canada, France, Germany, Hungary, Italy, Lithuania, Russia, Spain, UK and USA.
For further information see the clinical trial registration

*EHDN endorsement: The EHDN endorses protocols it finds to be of a high scientific and ethical quality. The endorsement decision is made on the basis of protocol review and discussion by the EHDN Executive Committee following recommendations from the independent EHDN Scientific and Bioethical Advisory Committee. Trial protocol endorsement should not be viewed as sponsor promotion or recruitment incitement. [EHDN endorsed studies overview]