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A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12- 17) with Phenylketonuria
EU CTIS ID: 2024-510875-39-00
What this study is testing
1 - To evaluate the efficacy of pegvaliase in adolescent participants with PKU 2 - To evaluate the safety of pegvaliase in adolescent participants with PKU
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Is 12 to 17 years old (US), inclusive, and 12 to 15 years old, (ex-US), 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 (defined as failed both sapropterin dihydrochloride treatment and Phe-restricted diet) demonstrated by 2 blood Phe concentration measurements > 600 µmol/L during the Screening/Run-in Period (7 to 10 days in between blood Phe assessments) and average blood Phe concentration > 600 µmol/L over the past 12 months (per available data).
- Willing and able to maintain and adjust dietary and medical protein food intake according to the study protocol and under the supervision of study dietician or adequately trained designee per investigator during study participation.
- If on medication for ADHD, depression, or other psychiatric disorder, stable dose of medication for ≥ 8 weeks prior to enrollment and willing to maintain stable dose unless a change is medically indicated.
- An adult (≥ 18 years of age) has been identified who is willing and competent to observe the participant during study drug administration and for a minimum of 1 hour following administration.
- Able and willing to comply with all study procedures.
You likely can't join if
- Have a history of a severe systemic hypersensitivity reaction or recurrence of a mild to moderate acute systemic hypersensitivity reaction to any of the excipients or another PEGylated medicinal product.
- Taking any of the prohibited medications outlined in Section 6.8.1.
- Previous treatment with pegvaliase.
- Use of any medication that is intended to treat PKU, including the use of large neutral amino acids, within 14 days prior to the administration of study drug on Day 1.
- Use or planned use of any injectable drugs containing polyethylene glycol (PEG; other than pegvaliase), including medroxyprogesterone injection, within 3 months prior to the start of Screening/Run-in and during study participation with the exception of COVID-19 vaccinations (Section 6.8.1).
- A history of organ transplantation or on chronic immunosuppressive therapy.
See the full eligibility criteria
Who can join
- Is 12 to 17 years old (US), inclusive, and 12 to 15 years old, (ex-US), 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 (defined as failed both sapropterin dihydrochloride treatment and Phe-restricted diet) demonstrated by 2 blood Phe concentration measurements > 600 µmol/L during the Screening/Run-in Period (7 to 10 days in between blood Phe assessments) and average blood Phe concentration > 600 µmol/L over the past 12 months (per available data).
- Willing and able to maintain and adjust dietary and medical protein food intake according to the study protocol and under the supervision of study dietician or adequately trained designee per investigator during study participation.
- If on medication for ADHD, depression, or other psychiatric disorder, stable dose of medication for ≥ 8 weeks prior to enrollment and willing to maintain stable dose unless a change is medically indicated.
- An adult (≥ 18 years of age) has been identified who is willing and competent to observe the participant during study drug administration and for a minimum of 1 hour following administration.
- Able and willing to comply with all study procedures.
- Generally in good health, as evidenced by physical examination, clinical laboratory evaluations (hematology, chemistry, and urinalysis), and electrocardiogram (ECG) tests performed at Screening.
- Males and females are eligible to participate in this clinical study.
- Females with childbearing potential must have a negative pregnancy test at Screening/Run-in and be willing to have additional pregnancy tests during the study.
What rules you out
- Have a history of a severe systemic hypersensitivity reaction or recurrence of a mild to moderate acute systemic hypersensitivity reaction to any of the excipients or another PEGylated medicinal product.
- Taking any of the prohibited medications outlined in Section 6.8.1.
- Previous treatment with pegvaliase.
- Use of any medication that is intended to treat PKU, including the use of large neutral amino acids, within 14 days prior to the administration of study drug on Day 1.
- Use or planned use of any injectable drugs containing polyethylene glycol (PEG; other than pegvaliase), including medroxyprogesterone injection, within 3 months prior to the start of Screening/Run-in and during study participation with the exception of COVID-19 vaccinations (Section 6.8.1).
- A history of organ transplantation or on chronic immunosuppressive therapy.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.