NCT01819727
Phenylketonuria
The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.
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.
All
18 Years to 70 Years
No
BMN 165
Phase 3
Interventional
261
2013-05
2019-02-26
La Jolla, California, United States
Oakland, California, United States
Aurora, Colorado, United States
Gainesville, Florida, United States
Miami, Florida, United States
Tampa, Florida, United States
Decatur, Georgia, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Lexington, Kentucky, United States
Louisville, Kentucky, United States
Boston, Massachusetts, United States
Detroit, Michigan, United States
Columbia, Missouri, United States
Saint Louis, Missouri, United States
Omaha, Nebraska, United States
Camden, New Jersey, United States
Morristown, New Jersey, United States
Albany, New York, United States
New York, New York, United States
Rochester, New York, United States
Cleveland, Ohio, United States
Oklahoma City, Oklahoma, United States
Portland, Oregon, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Nashville, Tennessee, United States
Houston, Texas, United States
Salt Lake City, Utah, United States
Seattle, Washington, United States
Milwaukee, Wisconsin, United States
INCLUSION CRITERIA
Individuals eligible to participate in this study must meet all of the following criteria:
* A current diagnosis of PKU with the following:
* Current blood Phe concentration >600 µmol/L at screening and
* Average blood Phe concentration of >600 µmol/L over the past 6 months (per available data)
* Have no previous exposure to BMN 165
* Are ≥18 and ≤70 years of age at the time of screening
* Subjects who are 1.5 times the upper limit of normal.
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