Roche Phase 3 Clinical Trial for Huntington’s Disease Gene-Silencing Drug to Enroll Volunteers – Early 2019

Dear friends in the fight against Huntington’s disease,

Pharma giant Roche expects to start enrolling subjects worldwide (including the U.S.) in early 2019 in its historic Phase 3 clinical trial of RG6042, a gene-silencing drug aimed at slowing, halting, and perhaps even reversing HD.

Roche made the announcement today at an HD research conference in Vienna, Austria, and issued a statement to the global HD community with details.

Designed by Roche partner Ionis Pharmaceuticals, Inc., RG6042 demonstrated impressive results in the Phase 1/2a trial completed by Ionis in December 2017.

In March 1, researchers revealed that RG6042 caused trial volunteers to experience a drop of 40 to 60 percent in the harmful mutant Huntington protein in their cerebral spinal fluid. However, this trial did not measure actual efficacy – only safety and tolerability.

Because of the solid Phase 1/2a results, Roche has taken the unusual step of skipping a Phase 2 trial (testing efficacy for the first time) and going directly to a robust Phase 3, where researchers hope to test efficacy in 660 volunteers over 25 months at 80 to 90 sites in approximately 15 countries.

Called GENERATION HD1, this trial will gauge the effects of reducing mutant Huntington and whether RG6042 can slow or stop the progression of HD. It will also further examine the drug’s safety.

This is yet another major step in the quest to treat HD!

You can read more about this exciting moment in “Roche Phase 3 clinical trial of Huntington’s disease gene-silencing drug to enroll volunteers in early 2019,” the latest article in my blog At Risk for Huntington’s Disease, which you can view at

As always, your feedback is welcome. You can post comments on the blog. You can also write me at [email protected]. I’m on Facebook (gene.veritas) and Twitter (@GeneVeritas), too.

Yours in the struggle to cure diseases,

Gene Veritas (aka Kenneth P. Serbin)