New study tracks PKU drug palynziq in pregnancy: what are the risks?

NCT ID NCT05579548

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 50 pregnant women with phenylketonuria (PKU) who have taken the drug Palynziq (pegvaliase) around the time of pregnancy or while breastfeeding. Researchers will track pregnancy outcomes and infant health, including birth defects and development. The goal is to gather real-world safety data, not to test a new treatment.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Pegvaliase (Palynziq)

What this could lead to

If successful, this study could provide crucial safety information for pregnant women with PKU who need Palynziq, helping doctors and patients make informed decisions.

What could go wrong

This is an observational study, not a controlled trial, so it cannot prove cause and effect. The small size (50 participants) limits how much we can learn, and results may not apply to all patients.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

maternal phenylketonuria phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Metabolics and Genetics in Calgary (MAGIC)

    RECRUITING

    Calgary, T2E 7Z4, Canada

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Ospedale San Paolo

    RECRUITING

    Milan, 20142, Italy

    Contact Email: •••••@•••••

  • Syneos Health

    RECRUITING

    Wilmington, North Carolina, 28403, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Universitaetsklinikum Hamburg Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

    Contact Email: •••••@•••••