Phase 4 Phenylketonuria Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are between 18 and 65 years old
- You have been formally diagnosed with phenylketonuria (PKU)
- You are generally in good health based on a medical check-up
- You are willing and medically suitable to take both Palynziq and methotrexate (MTX) during the study
- (Cohort A) You have never taken Palynziq before and are willing to start it as part of this study
- (Cohort B) You have been taking Palynziq for at least 24 weeks at a daily dose of at least 20mg, your blood PKU levels are still above a certain threshold, and your dose cannot be increased further (confirm with trial site)
- You agree to use the required birth control method if you or your partner could become pregnant
- You are willing to carry two epinephrine (severe allergic reaction) devices with you at all times while taking Palynziq
Who may not be able to join:
- You are pregnant, breastfeeding, planning to become pregnant, planning to father a child, or not using effective birth control if required
- You have a known serious allergy to methotrexate, Palynziq, or any medications containing PEG (a type of chemical ingredient)
- You have an active serious infection, or a history of severe or frequently recurring infections
- You have significant health conditions affecting your heart, lungs, liver, kidneys, immune system, nervous system, mental health, or have a history of cancer
- You have had issues with substance or alcohol misuse within the last 12 months
- You have had an organ transplant or regularly take medications that suppress your immune system
- You are currently taking medications that are not permitted in this study, including any PKU treatments other than Palynziq
- You are using, or plan to use, any other injectable medications containing PEG during the study
- You have a major surgery planned during the time you would be participating in the study
- You are already taking part in another clinical trial that involves Palynziq
- The study doctor feels you may not be a suitable candidate or may find it difficult to follow the study requirements
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Medical Director, MD, BioMarin Pharmaceutical
Phone: 1-800-983-4587
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Change in blood Phe levels from baseline to Week 25 and Week 49; Anti-PEG IgG, IgM, anti-PAL IgG, IgM levels at Weeks 5, 9, 25, and 49
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.