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

• Status: Completed
• Trial end date: 2021-01-13
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioMarin Pharmaceutical

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.

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.

• Current participation in the Kuvan® registry study (PKU Demographics, Outcomes and Safety [PKUDOS])

• A history of organ transplantation or on chronic immunosuppressive therapy