The PIVOT-HD study is a Phase II trial looking at how safety is PTC518, a new Huntington-Lowering drug, in patients with HD. PTC518 is an oral tablet (taken by mouth every day) that not only tries to lower huntingtin (mHTT) in the brain but also treat HD everywhere in the body (brain, muscle, immune system…).
This Phase 2 is hoping to enrol 162 participants. 2 doses will be given and also a placebo group over 12 weeks and a follow-up of 12 months.