Summary:
Prism 301: A Clinical Research Study for Adults with Phenylketonuria (PKU)
What is Phenylketonuria (PKU)?
People with phenylketonuria (PKU) can't process one of the amino acids found in many foods. The amino acid, called phenylalanine or "Phe" for short, builds up in the body. There are approximately 16,000 adults living with PKU in the United States and Canada.
Why is the Prism 301 study being done?
The main purpose of thePrism 301 study is to see how safe and tolerable an experimental enzyme substitution therapy called BMN 165 (also known as PEGPAL) is for adults with PKU.
What is PEGPAL?
PEGPAL is an experimental enzyme substitution therapy. “Experimental” means that it is not approved by the U.S. Food and Drug Administration (FDA) or Health Canada for the treatment of PKU. Study participants will be randomly assigned to one of two dosing groups. All study participants will receive PEGPAL.
Will I have to change my diet?
No. You will be asked to stay on your current diet during the study unless otherwise instructed by your study doctor.
How long will the study last?
Your participation in the study will last about 15 to 44 weeks. During the study, you will go to up to 14 study visits.
What will I be asked to do?
If you qualify for and decide to take part in the study, you will need to:
- Come to the study clinic for appointments
- Give yourself injections of PEGPAL as instructed by the study doctor
- Have blood and urine tests and other procedures at the study clinic
- Keep a diet diary for three days before certain tests
- Participate in weekly check-in phone calls with study staff
At the end of this study, you may have the option to participate in another PEGPAL study. The study doctor will provide you with details.
What are the potential risks and benefits?
The study doctor will explain all of the potential risks and benefits of participating in the study. Although you may or may not benefit from being in the study, the information learned from the study and your participation may help improve treatment options for people with PKU in the future.
Does it cost me anything to participate?
You will receive PEGPAL and study-related tests and procedures at no charge. In addition, you may receive reimbursement for your travel expenses.
Who can participate?
You may be eligible to participate in the study if you:
- Are 18 to 70 years old
- Have a diagnosis of PKU with a high Phe level (so that we can see if PEGPAL works to lower your Phe level)
- Have not received PEGPAL
Your eligibility will need to be confirmed at a study screening visit. If you are currently taking KUVAN®, please continue to follow your treatment plan as prescribed and talk to your PKU doctor.
Study participation is voluntary. If you choose to participate, you may leave the study at any time for any reason. The study doctor and staff will be available to help you throughout the study and to address any questions you may have. Your health is very important to them.
To find out if you may be eligible to participate, visit the Study Website .
Click here for an updated list of study sites.