Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
Prism-2
A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)
1 other identifier
interventional
215
1 country
29
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jul 2013
Longer than P75 for phase_3
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 18, 2013
CompletedFirst Posted
Study publicly available on registry
July 1, 2013
CompletedStudy Start
First participant enrolled
July 29, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 13, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
February 5, 2019
CompletedResults Posted
Study results publicly available
July 12, 2019
CompletedMay 21, 2021
April 1, 2021
2.5 years
June 18, 2013
June 22, 2018
April 29, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2
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.
At Part 2 baseline and change from baseline at Part 2 week 8
Secondary Outcomes (5)
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
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4
At Part 4 week 57, 105, 161, 209 and 249
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
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
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
Study Arms (4)
BMN165 20mg/day
ACTIVE COMPARATORBMN165 20mg/day self-administered daily
20 mg/day Placebo
PLACEBO COMPARATOR20 mg/day Placebo self-administered daily
BMN165 40mg/day
ACTIVE COMPARATORBMN165 40mg/day self-administered daily
40 mg/day Placebo
PLACEBO COMPARATOR40 mg/day Placebo self-administered daily
Interventions
Non-drug placebo, self-administered daily
Eligibility Criteria
You may qualify if:
- Individuals eligible to participate in this study must meet all of the following criteria:
- Have completed a prior BMN 165 study (PAL-003, 165-205, or 165-301) prior to screening
- Have had a stable BMN 165 dose regimen for at least 14 days prior to screening
- Are at least 18 y/o and no older than 70 y/o at screening
- Subjects who are \< 18 y/o and are already enrolled into Study 165-301 under Amendment #1 (10JAN2014) may enroll into this study
- Has identified a person who is \> 18 y/o who has the neurocognitive and linguistic capacities to comprehend and complete the Profile of Mood States (POMS)-Observer rated scale
- Has identified a competent person(s) \> 18 y/o who can observe the subject during study drug administration at certain points in the study
- A home healthcare nurse may perform the study drug observations
- Are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures; for minors, parent or guardian provides written consent and assent may be requested
- Are willing and able to comply with all study procedures
- For females of childbearing potential, a negative pregnancy test at screening and willing to have additional pregnancy tests during the study
- If sexually active, willing to use two acceptable methods of contraception during and for 4 weeks after the study
- Males post vasectomy for 2 years with no known pregnancies do not need to use any other forms of contraception during the study.
- Females who have been in menopause for at least 2 years, have had a tubal ligation at least 1 year prior to screening, or have had a total hysterectomy do not need to use any other forms of contraception during the study.
- Have received documented approval from a study dietitian confirming that the subject is capable of maintaining their diet
- +3 more criteria
You may not qualify if:
- Use of any investigational product (except BMN 165) or investigational medical device within 30 days prior to screening or requirement for any investigational agent prior to completion of all scheduled study assessments
- Use of any medication (except BMN 165) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of study drug
- Have known hypersensitivity to Dextran® or components of Dextran
- Use or planned use of any injectable drugs containing PEG (except for BMN 165), including medroxyprogesterone injection, within 3 months prior to screening and during study participation
- Current use of levodopa
- A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody
- A history of organ transplantation or taking chronic immunosuppressive therapy
- A history of substance abuse in the past 12 months or current alcohol or drug abuse
- Current participation in the Kuvan registry study (PKUDOS). Patients may discontinue the PKUDOS registry trial to allow enrollment in this study
- Pregnant or breastfeeding at screening or planning to become pregnant (self or partner) or breastfeed at any time during the study
- Concurrent disease or condition that would interfere with study participation or safety.
- Major surgery planned during the study period
- Any condition that in the view of the investigator, places the subject at high risk of poor treatment compliance or terminating early from the study
- Alanine aminotransferase (ALT) concentration at least 2x the upper limit of normal
- Creatinine at least 1.5x the upper limit of normal
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
University of California, San Diego Clinical and Translational Research Institute
La Jolla, California, 92093, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
University of South Florida
Tampa, Florida, 33606, United States
Emory Universty, Department of Human Genetics, Division of Medical Genetics
Atlanta, Georgia, 30322, United States
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Indiana CTSI Clinical Research Center
Indianapolis, Indiana, 46202, United States
University of Kentucky Medical Center
Lexington, Kentucky, 40536, United States
University of Louisville, Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Wayne State University Clinical Research Center at the Mott Center
Detroit, Michigan, 48202, United States
University of Missouri Health Center
Columbia, Missouri, 65212, United States
Washington University Center for Applied Research Sciences
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Cooper Health Systems
Camden, New Jersey, 08103, United States
Atlantic Health System - Morristown Medical Center
Morristown, New Jersey, 07960, United States
Albany Medical Center
Albany, New York, 12208, United States
Icahn School of Medicine at Mount Sinai Medical Center
New York, New York, 10029, United States
University Hospitals Case Medical Center
Cleveland, Ohio, 44106, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134, United States
Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232, United States
University of Texas Houston Medical School
Houston, Texas, 77030, United States
University of Utah Hospital
Salt Lake City, Utah, 84108, United States
University of Washinton
Seattle, Washington, 98195, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Related Publications (1)
Bilder DA, Arnold GL, Dimmock D, Grant ML, Janzen D, Longo N, Nguyen-Driver M, Jurecki E, Merilainen M, Amato G, Waisbren S. Improved attention linked to sustained phenylalanine reduction in adults with early-treated phenylketonuria. Am J Med Genet A. 2022 Mar;188(3):768-778. doi: 10.1002/ajmg.a.62574. Epub 2021 Nov 26.
PMID: 34826353DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Debra Lounsbury/Sr Principle Scientist, Clinical Sciences
- Organization
- BioMarin Pharmaceutical Inc
Study Officials
- STUDY DIRECTOR
Clinical Trial Specialist
BioMarin Pharmaceutical
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 18, 2013
First Posted
July 1, 2013
Study Start
July 29, 2013
Primary Completion
January 13, 2016
Study Completion
February 5, 2019
Last Updated
May 21, 2021
Results First Posted
July 12, 2019
Record last verified: 2021-04
Data Sharing
- IPD Sharing
- Will not share