Status:
ACTIVE_NOT_RECRUITING
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonuria (PKU)
Eligibility:
All Genders
12-17 years
Phase:
PHASE3
Brief 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), in...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
June 17 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2027
Estimated Enrollment :
55 Patients enrolled
Trial Details
Trial ID
NCT05270837
Start Date
June 17 2022
End Date
October 1 2027
Last Update
September 12 2025
Active Locations (16)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
3
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
4
University of South Florida
Tampa, Florida, United States, 33606