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Pegvaliase for Phenylketonuria (PEGASUS Trial)
PEGASUS Trial Summary
This trial is testing a new medication for PKU in adolescents. It is designed to see if it is safe and effective.
PEGASUS Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowPEGASUS Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Side effects data
From 2021 Phase 3 trial • 37 Patients • NCT03694353PEGASUS Trial Design
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Who is running the clinical trial?
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- Your creatinine levels in your blood are higher than 1.5 times the normal upper limit.I haven't used any PEG-containing injectable drugs recently, except for COVID-19 vaccines.I have had an organ transplant or am on long-term immune-weakening medication.You have tested positive for HIV, hepatitis B, or hepatitis C.Your liver enzyme levels are more than double the normal range.I have been treated with pegvaliase before.I haven't taken PKU treatments or large neutral amino acids in the last 14 days.I am using two forms of birth control and do not plan to get pregnant or father a child while in the study.I've been on a stable dose of my psychiatric medication for at least 8 weeks and can keep it steady.I am able to understand and sign the consent form.I have someone 18 or older to watch me for at least 1 hour after I take the study drug.I am willing to follow a diet plan supervised by a dietician for the study.You are unable to tell others if you are having symptoms of a severe allergic reaction.I have PKU and my blood Phe levels have been too high despite my current treatment.I am between 12 and 17 years old if in the US, or 12 to 15 if in the EU.
- Group 1: Drug: Diet Only
- Group 2: Pegvaliase
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
When was Pegvaliase cleared by the FDA?
"Pegvaliase has been shown to be effective in Phase 3 trials and has had multiple rounds of data supporting its safety, so it is estimated to be a safe medication."
How many individuals are being studied in this clinical trial?
"In order to move forward, this study requires 54 willing and eligible patients. If you qualify and are interested, know that you can participate in this trial at The Community Health Clinic in Topeka, Indiana and McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas."
Will elderly patients be accepted into this clinical trial?
"From the eligibility requirements, it can be inferred that the ideal participants for this trial fall between the ages of 12 to 17. Out of the 16 available clinical trials, 13 are for people over the age of 65."
Is this research being conducted in numerous hospitals across Canada?
"The Community Health Clinic in Topeka, Indiana, McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) in Houston, Texas, and University of Virginia School of Medicine in Charlottesville, Virginia are a few of the 20 active sites for this clinical trial."
Could you explain the inclusion criteria for this trial?
"This study is accepting 54 people with phenylketonurias aged 12 and 17. Most notable, applicants are required to meet the following criteria: Willing and able to maintain and adjust dietary and medical protein food intake according to the study protocol under the supervision of a study dietician or adequately trained designee per investigator discretion during study participation., Is 12 to 17 years old (US), inclusive, or 12 to 15 years (EU), inclusive, at the start of the Screening/Run-in Period (Day -28)., Diagnosis of PKU and failure to maintain recommended blood Phe levels on existing management ("
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