Pegvaliase + Methotrexate for PKU
What You Need to Know Before You Apply
What is the purpose of this trial?
Study 165-401 is a Phase 4, open-label study designed to examine the concomitant use of methotrexate (MTX) to suppress immune responses to Palynziq and improve tolerability and efficacy in adults with PKU.
Who Is on the Research Team?
Medical Director, MD
Principal Investigator
BioMarin Pharmaceutical
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
MTX Tolerability Period
Participants receive MTX to assess tolerability before combination treatment
Combination Treatment Period
Participants receive concurrent MTX and Palynziq treatment
Follow-up Period
Participants continue with Palynziq only and are monitored for safety and effectiveness
What Are the Treatments Tested in This Trial?
Interventions
- Methotrexate
- Pegvaliase
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Individuals with uncontrolled blood phenylalanine (Phe) \> 600 μmol/L on existing management who have never been treated with Palynziq. Participants will have a 4-week Screening Period, followed by a 4-week MTX Tolerability Period during which only MTX will be administered. If MTX is tolerated during the MTX Tolerability Period, the participant will then enter the 24-week Combination Treatment Period during which MTX + Palynziq will be administered concurrently. After completion of Week 24 (Combination Treatment Period), MTX will be discontinued for both cohorts and Palynziq only will continue for another 24 weeks (to Week 49).
Individuals who have uncontrolled blood Phe \> 600 μmol/L after ≥ 24 consecutive weeks of treatment with Palynziq, are on a current dose of ≥ 20mg/day and are unable to further dose escalate (Palynziq experienced). Participants will have a 4-week Screening Period. Participants will then have 24 weeks of concurrent MTX + Palynziq treatment (Combination Treatment Period): participants will be assessed for MTX tolerability during the first 4 weeks of MTX + Palynziq (MTX Tolerability Period) and then receive another 20 weeks of MTX + Palynziq. After completion of Week 24 (Combination Treatment Period), MTX will be discontinued for both cohorts and Palynziq only will continue for another 24 weeks (to Week 49).
Find a Clinic Near You
Who Is Running the Clinical Trial?
BioMarin Pharmaceutical
Lead Sponsor
Alexander Hardy
BioMarin Pharmaceutical
Chief Executive Officer since 2023
MBA from INSEAD
Greg Friberg
BioMarin Pharmaceutical
Chief Medical Officer
MD from New York Medical College
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