Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Drug: BMN165 40mg/dayDrug: BMN165 20mg/dayDrug: Placebo
- Registration Number
- NCT01889862
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief 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 adults with PKU.
- Detailed Description
165-302 is a Phase 3, 4 Part, Randomized, Double-Blind, Placebo-Controlled, Discontinuation Study to Evaluate the Efficacy and Safety of Self Administered Subcutaneous Injections of BMN 165 by Adults With PKU.
This study is open only to adults who have been exposed to BMN165 in a previous study.
Study BMN 165-302 is a four-Part, Phase 3 study.
* Part 1: Open-label period
* Part 2: A Randomized, double-blind, placebo-controlled period of 8 weeks
* Part 3: Two sub-Parts to Part 3:- Part 3A - BMN 165 administered using vial and syringe during this open label period. Part 3B - BMN 165 administered using prefilled syringe during this open-label period.
* Part 4: A long-term, open-label extension period.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 215
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description BMN165 40mg/day BMN165 40mg/day BMN165 40mg/day self-administered daily BMN165 20mg/day BMN165 20mg/day BMN165 20mg/day self-administered daily 40 mg/day Placebo Placebo 40 mg/day Placebo self-administered daily 20 mg/day Placebo Placebo 20 mg/day Placebo self-administered daily
- Primary Outcome Measures
Name Time Method Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 At Part 2 baseline and change from baseline at Part 2 week 8 Blood phe concentration in subjects previously exposed to BMN165 who were administered BMN165 20 or 40 mg/day compared with those who were administered a matching placebo.
- Secondary Outcome Measures
Name Time Method Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2 At Part 2 baseline and change from baseline at Part 2 week 8 Attention deficit hyperactivity disorder rating scale IV (ADHD RS-IV) is investigator-rated, 9-item, inattention subscale score ranges from 0 to 27, with higher scores indicative of a greater degree of impairment and a baseline score \>9 indicative of a presence of symptoms.
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4 At Part 4 week 57, 105, 161, 209 and 249 The long term effect of multiple dose levels of BMN165 on blood phe concentration in subjects who are administered BMN165 using pre-filled syringes.
Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS TMD at Part 2 At Part 2 baseline and change from baseline at Part 2 week 8 Phenylketonuria (PKU) Total Mood Disturbance (TMD) Profile of Mood States (POMS)
PKU POMS TMD is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely).Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS Confusion Subscale at Part 2 At Part 2 baseline and change from baseline at Part 2 week 8 PKU POMS confusion mood domain (referred to as the confusion subscale) is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely).
Change From Baseline in the Cognitive and Mood Symptoms Measured by POMS TMD at Part 2 At Part 2 baseline and change from baseline at Part 2 week 8 Full-length POMS TMD is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely).
Trial Locations
- Locations (29)
University of South Florida
🇺🇸Tampa, Florida, United States
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
Cooper Health Systems
🇺🇸Camden, New Jersey, United States
Vanderbilt Children's Hospital
🇺🇸Nashville, Tennessee, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Emory Universty, Department of Human Genetics, Division of Medical Genetics
🇺🇸Atlanta, Georgia, United States
Ann and Robert H Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center
🇺🇸Pittsburgh, Pennsylvania, United States
University of California, San Diego Clinical and Translational Research Institute
🇺🇸La Jolla, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
University of Florida Clinical Research Center
🇺🇸Gainesville, Florida, United States
Indiana CTSI Clinical Research Center
🇺🇸Indianapolis, Indiana, United States
University of Kentucky Medical Center
🇺🇸Lexington, Kentucky, United States
University of Louisville, Kosair Charities Pediatric Clinical Research Unit
🇺🇸Louisville, Kentucky, United States
Wayne State University Clinical Research Center at the Mott Center
🇺🇸Detroit, Michigan, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Missouri Health Center
🇺🇸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
Icahn School of Medicine at Mount Sinai Medical Center
🇺🇸New York, New York, United States
Atlantic Health System - Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
Albany Medical Center
🇺🇸Albany, New York, United States
University Hospitals Case Medical Center
🇺🇸Cleveland, Ohio, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
University of Texas Houston Medical School
🇺🇸Houston, Texas, United States
University of Utah Hospital
🇺🇸Salt Lake City, Utah, United States
University of Washinton
🇺🇸Seattle, Washington, United States
St. Christopher's Hospital for Children
🇺🇸Philadelphia, Pennsylvania, United States