Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

Running, not enrolling · Phase 3

Conditions studied: Phenylketonuria (PKU)

In brief

This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.

Key facts

Study ID

NCT05270837

Run by

BioMarin Pharmaceutical

People needed

55

Starts

2022-06-17

Expected to finish

2027-10-01

Last updated by the study team

2026-07-21

Who can join

Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 (sapropterin dihydrochloride 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 under the supervision of a study dietician or adequately trained designee per investigator discretion 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.
• Participants must be capable of giving signed informed consent
• If sexually active, male or female participants must not plan to become pregnant (self or partner) and must use 2 acceptable methods of contraception while participating in the study beginning at Screening and for 4 weeks after discontinuing study drug.

You may not qualify if…

• 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.
• A history of organ transplantation or on chronic immunosuppressive therapy.
• Use of any investigational product or investigational medical device within 30 days prior to Screening/Run-in or requirement for any investigational agent prior to completion of all scheduled study assessments.
• A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody.
• Alanine aminotransferase (ALT) concentration > 2 × the upper limit of normal (ULN).
• Creatinine > 1.5 × ULN.
• Inability to identify and/or communicate to others that the participant is experiencing symptoms of potential anaphylaxis due to cognitive impairment or other reasons.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• University of South Florida — Tampa, Florida, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• IU Health University Hospital — Indianapolis, Indiana, United States
• University of Kentucky College of Medicine — Lexington, Kentucky, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• McGovern Medical School, University of Texas Health Science Center at Houston (UTHealth) — Houston, Texas, United States
• University of Utah Medical Center — Salt Lake City, Utah, United States
• University of Virginia School of Medicine — Charlottesville, Virginia, United States
• Children's Hospital of Wisconsin — Milwaukee, Wisconsin, United States
• Charité - Universitätsmedizin Berlin — Berlin, Germany
• Universitätsklinikum Hamburg-Eppendorf — Hamburg, Germany
• Universitat Mainz — Mainz, Germany

Full record on ClinicalTrials.gov

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