Study aims and goal: The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 and placebo in participants with HD.
No. of study participants and sites: This phase IIa study aims to recruit participants from sites in Australia, France, Germany, Netherlands, the UK and the US.
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]