Phenylketonuria (PKU) MedDRA version: 20.0 Level: LLT Classification code 10034873 Term: Phenylketonuria (PKU) System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Palynziq
Product Name: Pegvaliase
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: Pegvaliase
CAS Number: 1585984-95-7
Current Sponsor code: pegvalias
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1 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). 2 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). 3 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. 4.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. 5.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. 6.Able and willing to comply with all study procedures. 7.Generally in good health, as evidenced by physical examination, clinical laboratory evaluations (hematology, chemistry, and urinalysis), and electrocardiogram (ECG) tests performed at Screening 8.Males and females are eligible to participate in this clinical study. 9.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. Are the trial subjects under 18? yes Number of subjects for this age range: 54 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. 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. 2. Taking any of the prohibited medications outlined in Section 6.8.1. 3.Previous treatment with pegvaliase. 4.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. 5.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). 6.A history of organ transplantation or on chronic immunosuppressive therapy.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: A- To evaluate the efficacy of pegvaliase in adolescent participants with PKU B - To evaluate the safety of pegvaliase adolescent participants with PKU ;Secondary Objective: A- To characterize total dietary protein intake in adolescent participants with PKU after pegvaliase treatment B- To characterize the pharmacokinetics (PK) of pegvaliase in adolescent participants with PKU ;Primary end point(s): Safety will be evaluated by assessment of AEs, SAEs, laboratory test results (chemistry, hematology, and urinalysis), vital signs physical examination, ECG and anti-BMN165 immunogenicity test results ;Timepoint(s) of evaluation of this end point: After 72 weeks of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1- Change from baseline in total dietary protein intake (ie, medical food and/or intact food). 2-Plasma sample for trough PK ;Timepoint(s) of evaluation of this end point: After 72 weeks of treatment | — |
Countries
Germany, United States
Contacts
Public ContactClinical Trial Information
BioMarin Pharmaceutical Inc.
Outcome results
None listed