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Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE3

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

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 Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design

NCT05270837•BioMarin Pharmaceutical

View on ClinicalTrials.gov

Summary

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.

Detailed Description

This Phase 3 multicenter study is designed to evaluate the safety and efficacy of pegvaliase administered daily to adolescents (ages 12 to 17 years old (US), inclusive, and 12 to 15 years old (EU), inclusive 12-17) with phenylketonuria (PKU). Participants will be randomized in a 2:1 ratio to the active (pegvaliase) and control (diet-only) treatment arms, respectively, with 36 participants receiving pegvaliase and 18 participants managing their PKU with diet alone.

Eligibility

Age

12 - 17 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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.

Exclusion Criteria

•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.

Locations (16)

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

Key Dates

Start Date

June 17, 2022

Primary Completion Date

January 14, 2025

Completion Date

October 1, 2027

Last Updated

September 12, 2025

Enrollment

ESTIMATED participants

Conditions

Phenylketonuria (PKU)

Interventions

Pegvaliase

DRUG

Diet Only

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 12, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

AAV Gene Therapy Study for Subjects with PKU

A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study

PANDA: PKU Amino Acid Evaluation

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