“As time has gone on and we’ve understood more about the pharmacodynamics of the drug, we’ve been able to explore less frequent dosing regimens that we still believe will produce huntingtin-lowering in the range that’s likely to be effective.”

RG6042 is emerging as a possible treatment for Huntington disease, with positive first-in-human findings being presented earlier this year at the American Academy of Neurology Annual Meeting. Now, it is being assessed by developer Roche in a phase 3 trial, called GENERATION HD1 (NCT03761849).

Edward Wild, MBBCHir, PhD, MA, neurologist, Huntington’s Disease Center, University College London, told NeurologyLive that the first-in-human data “was incredibly gratifying, especially considering this was the first time the drug had been given to people.” When asked more about the next step for the therapy and what’s to be expected from the GENERATION HD1 study, Wild shared further insight into the trial design and the unique nature of RG6042’s dosing.

The global study originally planned to enroll up to 660 patients with manifest HD at 80 to 90 sites in approximately 15 countries around the world, but will now enroll up to 801 patients in more than 90 sites in 19 countries around the world, with the hope to provide more data to equally asses both dosing groups and improve the statistical power of the study. More information on the clinical trial sites can be found here