RecruitingOBSERVATIONAL

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

This study is gathering information about women with a genetic condition called Phenylketonuria (PKU) who are treated with a medicine called Palynziq (pegvaliase) and become pregnant or are breastfeeding. Researchers want to understand how taking Palynziq affects both the mother and her baby. It's an observational study, meaning doctors will collect information from existing medical records and during the pregnancy and the baby’s first year. This will help understand the effects of Palynziq exposure during this important time. The study will look at various health outcomes for both the mother and the baby to ensure the medicine is as safe as possible for everyone involved.

At a glance

Status

Recruiting

Sponsor

BioMarin Pharmaceutical

Enrolment target

Start

22 Nov 2022

Estimated completion

04 Oct 2032

Phenylketonuria, Maternal

What is this study about?

This research is collecting important information about pregnant women who have a condition called Phenylketonuria, or PKU, and are taking a medication known as Palynziq (pegvaliase). PKU is a rare genetic condition where the body can't break down a certain substance found in food. This can cause health problems if not managed well. Palynziq is a treatment that helps manage PKU.

The main goal of this study is to find out what happens to pregnant women with PKU who are taking Palynziq, and how their babies are affected, both during pregnancy and while they are breastfeeding. Doctors want to understand the outcomes for both mothers and their children when Palynziq is used during these crucial times. This is done by carefully observing and collecting health information from their medical records without giving any new treatments.

By gathering this detailed information, researchers hope to better understand the safety profile of Palynziq for pregnant and breastfeeding women with PKU and their babies. This knowledge is vital to help doctors and patients make the best choices about PKU treatment during pregnancy and breastfeeding in the future.

Key takeaways

This study focuses on pregnant women with PKU who take Palynziq.
It's an 'observational study' – no new treatments are given.
Data will be collected about mothers and babies during pregnancy and the baby's first year.
The goal is to understand Palynziq's effects during pregnancy and breastfeeding.
Participation lasts about 21 months.
Your medical information will be collected from your healthcare providers.

Who may be eligible?

This study is for women who are already pregnant and have Phenylketonuria (PKU). You must have been treated with the medicine Palynziq (pegvaliase) at some point, starting from at least two weeks before your last period. You also need to agree to let your doctors share your medical information with the study team. This includes information from your family doctor, your PKU specialist, your obstetrician (pregnancy doctor), and your baby’s doctor.

Before you can join, you or your legal representative must give your clear permission. This permission will follow all local rules and requirements. You cannot be currently involved in any other study sponsored by BioMarin that involves a new drug, device, or procedure.

This study is specifically for women and does not have an age limit, meaning it's open to all adult women who meet the other requirements.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you currently pregnant?
Have you been diagnosed with Phenylketonuria (PKU)?
Have you taken Palynziq (pegvaliase) at any point since two weeks before your last period?
Are you able to provide permission for your doctors to share your medical information?
Are you NOT currently in another BioMarin-sponsored drug or device study?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the first step is to contact a special call center. Once you agree to join, your doctors will start collecting information about your health. For example, they will look back at your medical records for at least three months before your last period to understand your health history and how you were using Palynziq.

Throughout your pregnancy, and then for your baby's first year of life, doctors will continue to collect information. This includes details about your use of Palynziq during each stage of your pregnancy and if you're breastfeeding. You won't be given any new medication for the study; doctors will just be observing and recording information from your standard medical care. Your involvement in the study will last for approximately 21 months in total.

Potential risks and benefits

The main benefit of joining this study is that it helps doctors learn more about how Palynziq affects pregnancy and breastfeeding in women with PKU. This information is really important and could help many other women and their babies in the future. Since this is an observational study, you will not be given any new treatments or procedures beyond your usual medical care, so there are no known direct medical risks from participating. However, there is a risk that your medical information will be shared with the study team, though this is done securely and confidentially. Remember, taking part is entirely voluntary, and you have the right to leave the study at any time without it affecting your medical care.

Locations (4)

Syneos Health
Verified postcode
Wilmington, United States· Recruiting
Metabolics and Genetics in Calgary (MAGIC)
Verified postcode
Calgary, Canada· Recruiting
Universitaetsklinikum Hamburg Eppendorf
Verified postcode
Hamburg, Germany· Recruiting
Ospedale San Paolo
Verified postcode
Milan, Italy· Recruiting

Common questions

What is PKU?

PKU, or Phenylketonuria, is a rare genetic condition where your body can't properly break down a substance found in many foods. If not managed, it can lead to health problems.

What is Palynziq (pegvaliase)?

Palynziq is a medicine used to treat PKU. It helps your body manage the substance it can’t break down properly.

Will I get new medicine in this study?

No, this study is about collecting information from your existing medical care. You won't be given any new medicine or treatments as part of the study.

How long will I be in the study?

If you join, your participation will last for about 21 months, covering your pregnancy and your baby's first year.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time. This will not affect your medical care in any way.

How to find out more

165-504 Program Director

medinfo@bmrn.com 1-800-983-4587 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.