Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
Condition: Phenylketonuria (PKU)
MeSH terms: Phenylketonurias
Intervention: Pegvaliase
Intervention description: 40-60mg/day
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 37.0
Sex: All
Minimum age: 18 Years
Maximum age: 70 Years
Healthy volunteers: No

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:

        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