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

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Trial Status Completed

Trial Identifier

NCT03694353

Condition

Phenylketonuria, Phenylketonuria (PKU)

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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.

Eligibility Criteria

Birth Sex

All

Age

18 Years to 70 Years

Healthy Volunteers

No

Drug/Treatment:

Pegvaliase

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

37

Study Started:

2018-09-13

Study Updated:

2022-02-17

Trial Locations

  • 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

    Saint 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

Eligibility Criteria

Inclusion criteria:

* 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).

Exclusion criteria:

* 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

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