BioMarin Pharmaceutical
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.
The overall objective of the study is to evaluate the ability of MTX, when co-administered with Palynziq, to suppress immune responses to Palynziq and thus improve the tolerability and efficacy of Palynziq in adults with phenylketonuria (PKU). Up to 12 participants will be enrolled into the study across 2 cohorts, with a target enrollment of 6 participants into each cohort: * Cohort A: Individuals with uncontrolled blood phenylalanine (Phe) \> 600 μmol/L on existing management who have never been treated with Palynziq (Palynziq naïve) * Cohort B: Individuals who have uncontrolled blood Phe \>…
Inclusion Criteria: * Adults between 18 and 65 years old * Have a confirmed diagnosis of phenylketonuria (PKU) * Are in generally good health based on medical evaluation * Are willing and medically eligible to receive Palynziq and methotrexate (MTX) Cohort A: Have never taken Palynziq before and are willing to start it during the study Cohort B: Have blood \> 600 μmol/L after taking Palynziq for at least 24 weeks, are on a daily dose of at least 20mg and unable to increase the dose further * Agree to use required contraception if they or their partner could become pregnant * Are willing to ca…
Pegvaliase (Palynziq) administered by subcutaneous injection. Dosing follows U.S. Prescribing Information and investigator judgement.
Oral methotrexate 15 mg administered once weekly during the Tolerability Period and the Combination Treatment Period.