An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
Public ClinicalTrials.gov record NCT01819727. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety & Tolerability of an Induction, Titration, and Maintenance Dose Regimen of BMN 165 Self Administered by Adults With PKU Not Previously Treated With BMN 165
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT01819727
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 261 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years to 70 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 2013 (Type not available)
- Primary completion
- Nov 25, 2015 (Actual)
- Completion
- Nov 25, 2015 (Actual)
- Last update posted
- Feb 26, 2019
2013 – 2015
United States locations
- U.S. sites
- 31
- U.S. states
- 22
- U.S. cities
- 31
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, Altman Clinical and Translational Research Institute | La Jolla | California | 92037 | — |
| UCSF Benioff Children's Hospital Oakland | Oakland | California | 94609 | — |
| The Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| University of Florida Clinical Research Center | Gainesville | Florida | 32610 | — |
| University of Miami Health System | Miami | Florida | 33136 | — |
| University of South Florida | Tampa | Florida | 33606 | — |
| Emory Universty | Decatur | Georgia | 30033 | — |
| Ann and Robert H Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Riley Children's Hospital | Indianapolis | Indiana | 46202 | — |
| University of Kentucky Medical Center | Lexington | Kentucky | 40536 | — |
| Weisskopf Child Evaluation Center / University of Louisville | Louisville | Kentucky | 40202 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Wayne State University | Detroit | Michigan | 48201 | — |
| University of Missouri | Columbia | Missouri | 65212 | — |
| Washington University Center for Applied Research Sciences | St Louis | Missouri | 63110 | — |
| University of Nebraska Medical Center | Omaha | Nebraska | 68198 | — |
| Cooper Health Systems | Camden | New Jersey | 08103 | — |
| Atlantic Health System - Morristown Medical Center | Morristown | New Jersey | 07960 | — |
| Albany Medical College | Albany | New York | 12208 | — |
| Icahn School of Medicine at Mount Sinai Medical Center | New York | New York | 10029 | — |
| University of Rochester | Rochester | New York | 14642 | — |
| University Hospital Cleveland, Case Medical Center | Cleveland | Ohio | 44106 | — |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | — |
| Oregon Health & Science University | Portland | Oregon | 97239 | — |
| St. Christopher's Hospital for Children | Philadelphia | Pennsylvania | 19134 | — |
| Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15224 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| University of Texas Health Science at Houston | Houston | Texas | 77030 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
| University of Washington Medical Center | Seattle | Washington | 98195 | — |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01819727, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 26, 2019 · Synced Oct 17, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01819727 live on ClinicalTrials.gov.