An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

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Trial Status Completed

Trial Identifier

NCT01819727

Condition

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.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

18 Years to 70 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 165

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

261

Study Started:

2013-05

Study Updated:

2019-02-26

Trial Locations

  • University of California, Altman Clinical and Translational Research Institute

    La Jolla, California, United States

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, United States

  • The Children's Hospital Colorado

    Aurora, Colorado, United States

  • University of Florida Clinical Research Center

    Gainesville, Florida, United States

  • University of Miami Health System

    Miami, Florida, United States

  • University of South Florida

    Tampa, Florida, United States

  • Emory Universty

    Decatur, Georgia, United States

  • Ann and Robert H Lurie Children's Hospital of Chicago

    Chicago, Illinois, United States

  • Riley Children's Hospital

    Indianapolis, Indiana, United States

  • University of Kentucky Medical Center

    Lexington, Kentucky, United States

  • Weisskopf Child Evaluation Center / University of Louisville

    Louisville, Kentucky, United States

  • Boston Children's Hospital

    Boston, Massachusetts, United States

  • Wayne State University

    Detroit, Michigan, United States

  • University of Missouri

    Columbia, Missouri, United States

  • Washington University Center for Applied Research Sciences

    Saint Louis, Missouri, United States

  • University of Nebraska Medical Center

    Omaha, Nebraska, United States

  • Cooper Health Systems

    Camden, New Jersey, United States

  • Atlantic Health System - Morristown Medical Center

    Morristown, New Jersey, United States

  • Albany Medical College

    Albany, New York, United States

  • Icahn School of Medicine at Mount Sinai Medical Center

    New York, New York, United States

  • University of Rochester

    Rochester, New York, United States

  • University Hospital Cleveland, Case Medical Center

    Cleveland, Ohio, United States

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, United States

  • Oregon Health & Science University

    Portland, Oregon, United States

  • St. Christopher's Hospital for Children

    Philadelphia, Pennsylvania, United States

  • Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, United States

  • University of Texas Health Science at Houston

    Houston, Texas, United States

  • University of Utah

    Salt Lake City, Utah, United States

  • University of Washington Medical Center

    Seattle, Washington, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, United States

Eligibility Criteria

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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