Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00924703
• Date of registration: 18/06/2009
• Prospective Registration: Yes
• Primary sponsor: BioMarin Pharmaceutical
• Public title: Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
• Scientific title: Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKU
• Date of first enrolment: October 8, 2009
• Target sample size: 68
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00924703
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Medical Director, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion 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.



Age minimum: 16 Years
Age maximum: 55 Years
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Intervention(s)

Drug: rAvPAL-PEG

Primary Outcome(s)

Blood Phe concentrations [Time Frame: 3 days postdose]

Secondary Outcome(s)

Safety based on the incidence of adverse events and clinically significant changes in vital signs [Time Frame: Screening and monthly up to 98 months]

Pharmacokinetics [Time Frame: Week 1 and quarterly thereafter, up to Week 392 or Quarter 32]

Antibody response [Time Frame: Week 1 and quarterly thereafter (eg. Week 12, 24, etc.up to Week 392)]

Safety based on clinically significant changes in laboratory test results [Time Frame: Screening and monthly thereafter up to 98 Months]

Secondary ID(s)

PAL-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors