Phenylketonuria Trial, Recruiting NCT05579548 Sponsor: BioMarin Pharmaceutical Condition: Phenylketonuria
Back to Phenylketonuria

Phenylketonuria Trial, Recruiting

NCT05579548
Recruiting Not Applicable

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 (or someone legally authorized to speak for you) must agree to take part before joining, following any rules required in your country
  • You must currently be pregnant, or have recently been pregnant (including if the outcome of the pregnancy is not yet known, or if you have already had prenatal tests such as an ultrasound or amniocentesis)
  • You must have been diagnosed with PKU (phenylketonuria) according to your local doctor's standard practice
  • You must have been treated with a medication called pegvaliase at any point starting from 2 weeks before the first day of your last menstrual period
  • You must agree to allow the study team to contact your doctors, midwife, or nurses — and your baby's doctors — to collect medical information

Who may not be able to join:

  • You cannot currently be taking part in another clinical study sponsored by BioMarin that involves an investigational (experimental) medicine, device, or procedure

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.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

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

Status
Recruiting
Phase
Not Applicable
Sponsor
BioMarin Pharmaceutical
Registry
ClinicalTrials.gov
Start date
22 November 2022
Est. completion
4 October 2032

Where this trial is recruiting

🇨🇦 Canada 🇩🇪 Germany 🇮🇹 Italy 🇺🇸 United States

Primary endpoints

Pregnancy outcome and infant development for women who have been treated with Palynziq (pegvaliase) within 2 weeks of their last menstrual period

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

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.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov