Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

Completed · Phase 3

Conditions studied: Phenylketonuria (PKU)

In brief

This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570). The study is designed to evaluate the long-term safety and efficacy of pegvaliase administered at doses \> 40 mg/day to 60 mg/day.

Key facts

Study ID

NCT03694353

Run by

BioMarin Pharmaceutical

People needed

37

Starts

2018-09-13

Expected to finish

2021-01-13

Last updated by the study team

2022-02-17

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Must be enrolled in PAL-003 or 165-302 Part 4 at the time of screening for 165-304 and most recently receiving pegvaliase at a dose > 40 mg/day.
• Has identified a competent person or persons ≥ 18 years of age who can observe the subject during study drug administration and for a minimum of 1 hour following administration in situations required per protocol.
• For females with childbearing potential, must have negative pregnancy test at screening and be willing to have additional pregnancy tests during the study.
• If sexually active and not planning to become pregnant (self or partner), must be willing to use 2 acceptable methods of contraception while participating in the study and for 4 weeks after the study.
• Is 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; a legally authorized representative may provide written consent and assent may be requested.
• Is willing and able to comply with all study procedures.
• Is in generally good health, as evidenced by physical examination and/or clinical laboratory evaluations (hematology, chemistry, and urinalysis).

You may not qualify if…

• Use of any investigational product (except pegvaliase) 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 pegvaliase) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of pegvaliase (Day 1).
• Use or planned use of any injectable drugs containing PEG (other than pegvaliase), including medroxyprogesterone injection, within 3 months prior to screening and during study participation.
• A history of organ transplantation or on chronic immunosuppressive therapy
• A history of substance abuse (as defined by the American Psychiatric Association: Diagnostic and Statistical Manual of Mental Disorders [DSM]) in the past 12 months or current alcohol or drug abuse
• Current participation in the Kuvan® registry study (PKU Demographics, Outcomes and Safety [PKUDOS])
• Concurrent disease or condition that would interfere with study participation or safety (eg, history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric disease)
• 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

Where it is running

• University of Florida — Gainesville, Florida, United States
• University of South Florida — Tampa, Florida, United States
• Ann & 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
• Wayne State University, Center for Molecular Medicine and Genetics, Pediatrics and Pathology — Detroit, Michigan, United States
• University of Missouri Health Care — Columbia, Missouri, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• University of Nebraska Medical Center — Omaha, Nebraska, United States
• Morristown Medical Center — Morristown, New Jersey, United States
• University of Oklahoma Health Science 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
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• University of Texas Health Science Center at Houston — Houston, Texas, United States
• Medical College of Milwaukee — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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