Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
Condition: Phenylketonuria
MeSH terms: Phenylketonurias
Intervention: rAvPAL-PEG
Intervention description: The doses are planned to be in the same range as those tested in PAL-002 or PAL-004 and then modified either by increasing or decreasing the dose, adhering to an upper limit up to 5.0 mg/kg per week or 375 mg/week, considering each subject's individual responses related to safety and efficacy.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 68.0
Sex: All
Minimum age: 16 Years
Maximum age: 55 Years
Healthy volunteers: No

Summary:

      This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will
      be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain
      reduced blood Phe concentrations in PKU subjects.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Must have completed participation in previous rAvPAL-PEG studies.

          -  Willing and able to provide written, signed informed consent, or, in the case of
             participants under the age of 18, provide written assent (if required) and written
             informed consent by a parent or legal guardian, after the nature of the study has been
             explained, and prior to any research-related procedures.

          -  Willing and able to comply with all study procedures.

          -  Females of childbearing potential must have a negative pregnancy test at Screening and
             be willing to have additional pregnancy tests during the study. Females considered not
             of childbearing potential include those who have been in menopause at least 2 years,
             or had tubal ligation at least 1 year prior to Screening, or who have had total
             hysterectomy.

          -  Sexually active subjects must be willing to use an acceptable method of contraception
             while participating in the study.

          -  Maintained a stable diet.

          -  In generally good health as evidenced by physical examination, clinical laboratory
             evaluations (hematology, chemistry, and urinalysis), and electrocardiogram (ECG) at
             Screening.

        Exclusion Criteria:

          -  Use of any investigational product (with the exception of rAvPAL-PEG) 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 that is intended to treat PKU within 14 days prior to the
             administration of study drug.

          -  Use or planned use of any injectable drugs containing PEG (other than rAvPAL-PEG),
             including Depo-Provera during study participation.

          -  A prior reaction that included systemic symptoms (eg, generalized hives, respiratory
             or gastrointestinal problems, hypotension, angioedema, anaphylaxis) to rAvPAL-PEG or a
             PEG-containing product.

          -  Pregnant or breastfeeding at Screening or planning to become pregnant (self or
             partner) or to breastfeed at any time during the study.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 PI, places the subject at high risk of poor
             treatment compliance or of not completing the study.

          -  Known hypersensitivity to rAvPAL-PEG or its excipients, including hypersensitivity
             reactions that necessitated early termination from previous rAvPAL-PEG studies.

          -  Alanine aminotransferase (ALT) concentration > 2 times the upper limit of normal.

          -  Creatinine > 1.5 times the upper limit of normal.