PREMIER is a Phase 3 clinical study of a study drug called PXT3003. The study is sponsored by Pharnext.
Patient recruitment is now closed.
There is currently no approved medicine for CMT1A, and treatment consists of supportive care such as orthoses, physical and occupational therapy or surgery.
The purpose of the PREMIER study is to discover if PXT3003 is effective at treating CMT1A and to find out how safe it is to take. In the study, PXT3003 will be compared against a placebo (an inactive or “dummy” drug), so subjects in the study may end up receiving a placebo instead of PXT3003.