Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT ID: NCT01889862
Last Updated: 2021-05-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
215 participants
INTERVENTIONAL
2013-07-29
2019-02-05
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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BMN165 20mg/day
BMN165 20mg/day self-administered daily
BMN165 20mg/day
BMN165 20mg/day self-administered daily
20 mg/day Placebo
20 mg/day Placebo self-administered daily
Placebo
Non-drug placebo, self-administered daily
BMN165 40mg/day
BMN165 40mg/day self-administered daily
BMN165 40mg/day
BMN165 40mg/day self-administered daily
40 mg/day Placebo
40 mg/day Placebo self-administered daily
Placebo
Non-drug placebo, self-administered daily
Interventions
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BMN165 20mg/day
BMN165 20mg/day self-administered daily
BMN165 40mg/day
BMN165 40mg/day self-administered daily
Placebo
Non-drug placebo, self-administered daily
Other Intervention Names
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Eligibility Criteria
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Inclusion 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
* Have neurocognitive and linguistic capacities to comprehend and answer investigator's prompts for the Attention Deficit Hyperactivity Disorder Rating Scale (ADHD-RS) Investigator rated instrument and to complete the POMS-Subject rated scale
* If applicable, maintained stable dose of medication for Attention Deficit Hyperactivity Disorder (ADHD), depression, anxiety, or other psychiatric disorder ≥8 weeks prior to enrollment and willing to maintain stable dose throughout study unless a change is medically indicated
* General good health, as evidenced by physical examination, clinical laboratory evaluations, and ECG tests at screening
Exclusion Criteria
* 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
18 Years
70 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Specialist
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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University of California, San Diego Clinical and Translational Research Institute
La Jolla, California, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
University of South Florida
Tampa, Florida, 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
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
Boston Children's Hospital
Boston, Massachusetts, United States
Wayne State University Clinical Research Center at the Mott Center
Detroit, Michigan, United States
University of Missouri Health Center
Columbia, Missouri, United States
Washington University Center for Applied Research Sciences
St 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 Center
Albany, New York, United States
Icahn School of Medicine at Mount Sinai Medical Center
New York, New York, United States
University Hospitals Case Medical Center
Cleveland, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Oregon Health and Science University
Portland, Oregon, United States
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Vanderbilt Children's Hospital
Nashville, Tennessee, 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
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Countries
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References
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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.
Related Links
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BioMarin sponsored PKU educational/community website
Other Identifiers
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165-302
Identifier Type: -
Identifier Source: org_study_id
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