An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
Status:
Completed
Trial end date:
2015-11-25
Target enrollment:
Participant gender:
Summary
The BMN 165 clinical development program has been designed to demonstrate the safety and
efficacy of BMN 165 in reducing blood Phe concentrations in patients 18 to 70 years old with
hyperphenylalaninemia due to PKU. Study BMN 165-301 is a Phase 3, open-label, randomized
study designed to further characterize the safety of BMN 165 during two induction, titration,
and maintenance dose regimens in adults with PKU who have not had previous exposure to BMN
165 (naive). Subjects will be randomized (1:1) to titrate up to one of two dose regimens.
Other key features of this study are the dose regimens chosen for induction and titration;
the study duration; self administration of study drug; and the chosen tertiary objectives.