RecruitingOBSERVATIONAL

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

This is a 10-year study that watches people who are taking or are about to start a medicine called pegvaliase for their Phenylketonuria (PKU). Its main goal is to understand the long-term safety of pegvaliase, especially any allergic reactions or other side effects that might happen over time. Researchers also want to see how well certain safety measures are working, particularly in the European Union. This study isn't testing a new treatment; it's simply observing patients' experiences as they receive their usual care for PKU while on pegvaliase. It helps doctors and patients better understand the full picture of this treatment for PKU.

At a glance

Status

Recruiting

Sponsor

BioMarin Pharmaceutical

Enrolment target

450

Start

06 Jun 2022

Estimated completion

01 Nov 2033

Phenylketonuria (PKU)

What is this study about?

This study, called PALace, is all about understanding the long-term effects of a medicine known as pegvaliase, which is used to treat a condition called Phenylketonuria (PKU). PKU is a rare genetic condition where the body can't break down a substance called phenylalanine, which can build up and cause health problems. Pegvaliase helps to lower phenylalanine levels in the blood.

The study will follow people for 10 years to see if there are any side effects or health concerns that show up over a longer period. This includes looking at things like allergic reactions, general well-being, and how people feel over time while taking the medicine. It also checks how effective the extra safety steps put in place (especially in Europe) are at protecting patients.

It's important to remember that this is an 'observational' study. This means researchers are simply watching what happens in people's real lives as they take pegvaliase as part of their regular care. They are not asking anyone to try a new, untested treatment, nor are they changing how doctors would normally treat their patients. The aim is to gather real-world information to make sure pegvaliase is as safe and effective as possible for everyone who needs it.

Key takeaways

This study tracks the long-term safety of pegvaliase for people with PKU.
It lasts for 10 years and aims to identify any rare or long-term side effects.
No new treatments or extra appointments are required for participants.
Information is collected from your regular doctor visits.
The study helps improve understanding of pegvaliase for future patients.

Who may be eligible?

You might be able to join this study if you have been diagnosed with Phenylketonuria (PKU).

To be considered, you must either be already taking pegvaliase for your PKU, or your doctor has decided that you will start taking pegvaliase within 30 days of joining the study. This includes people who might have taken pegvaliase as part of a research program before.

You cannot take part if you are already in another study where you are receiving a new experimental treatment or procedure. Also, you shouldn't join if for any reason, your doctor thinks you wouldn't be able to understand the study information or follow what's asked of you.

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.

Do I have a confirmed diagnosis of Phenylketonuria (PKU)?
Am I currently taking pegvaliase, or will I start taking it within the next month?
Am I willing to allow my doctor to share information from my regular appointments for research?
Am I not currently taking part in any other study that involves new, experimental medicines or procedures?

Answer every question to see your result.

What does participation involve?

If you join this study, you won't need to have any extra visits or special tests just for the study. Instead, your own doctor (who is also the study doctor) will gather information from your regular check-ups and medical records. This includes details about your medical history, any other treatments you're receiving, and how your PKU is being managed with pegvaliase.

Throughout the study, your doctor will keep an eye out for specific things like allergic reactions, joint pain, reactions at the injection site, or if your phenylalanine levels get too low. These are all things doctors usually monitor anyway when someone is taking pegvaliase. Since the study lasts 10 years, your doctor will continue to collect this information during your routine appointments, typically every month when you first start pegvaliase and then every six months once your treatment is settled.

Potential risks and benefits

This study is observational, meaning you won't be given any experimental treatments and your normal care won't change. Therefore, there are no direct medical risks from participating in the study itself. The information gathered may help doctors and researchers better understand the long-term safety of pegvaliase, which could benefit future patients with PKU. You are free to withdraw from the study at any time without explaining why, and this will not affect your medical care.

Locations (26)

University of Colorado
Verified postcode
Aurora, United States· Recruiting
Emory University
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Atlanta, United States· Recruiting
Northwestern University
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Chicago, United States· Recruiting
Indiana University School of Medicine
Verified postcode
Indianapolis, United States· Recruiting
Tulane University Medical Center
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New Orleans, United States· Recruiting
Boston Children's Hospital
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Boston, United States· Recruiting
Oregon Health and Science University
Verified postcode
Portland, United States· Recruiting
University of Pittsburgh Medical Center
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Pittsburgh, United States· Recruiting
Vanderbilt University Medical School
Verified postcode
Nashville, United States· Recruiting
University of Texas Southwestern Medical Center
Verified postcode
Dallas, United States· Recruiting
University of Utah
Verified postcode
Salt Lake City, United States· Recruiting
Medical College of Wisconsin
Verified postcode
Milwaukee, United States· Recruiting

Common questions

What is Phenylketonuria (PKU)?

PKU is a rare condition where your body can't properly break down a substance called phenylalanine, which can then build up and cause health problems.

What is pegvaliase?

Pegvaliase is a medicine used to help lower phenylalanine levels in people with PKU.

Will I have to take new medicine or get extra tests for this study?

No, this study only watches what happens with your regular treatment and check-ups. You won't get any new medicines or extra tests just for the study.

How long will I be in this study?

The study collects information about patients for up to 10 years, but your doctor will gather this during your normal appointments.

Can I stop being part of the study at any time?

Yes, you can decide to leave the study whenever you want, and your medical care will not be affected.

How to find out more

165-501 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.