PEGVALIASE (pegvaliase) for Phenylketonuria

Inclusion criteria

• {"criterion_text":"- 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)."}
• {"criterion_text":"- 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)."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Able and willing to comply with all study procedures."}
• {"criterion_text":"- Generally in good health, as evidenced by physical examination, clinical laboratory evaluations (hematology, chemistry, and urinalysis), and electrocardiogram (ECG) tests performed at Screening."}
• {"criterion_text":"- Males and females are eligible to participate in this clinical study."}
• {"criterion_text":"- 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."}

Exclusion criteria

• {"criterion_text":"- 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."}
• {"criterion_text":"- Taking any of the prohibited medications outlined in Section 6.8.1."}
• {"criterion_text":"- Previous treatment with pegvaliase."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 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)."}
• {"criterion_text":"- A history of organ transplantation or on chronic immunosuppressive therapy."}

Primary endpoints

• {"endpoint_text":"- 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","definition_or_measurement_approach":"Safety assessed by monitoring adverse events (AEs), serious adverse events (SAEs), laboratory tests (chemistry, hematology, urinalysis), vital signs, physical examination, ECG, and anti-BMN165 (immunogenicity) test results."}

Secondary endpoints

• {"endpoint_text":"- Change from baseline in total dietary protein intake (ie, medical food and/or intact food).","definition_or_measurement_approach":"Measured as change from baseline in total dietary protein intake (including medical food and/or intact food) per available dietary assessments."}
• {"endpoint_text":"- Plasma sample for trough PK","definition_or_measurement_approach":"Collection of plasma samples at trough timepoints to characterize pharmacokinetics (PK) parameters."}

Germany

Earliest CTIS Part Ii Submission Date

16-04-2024

Latest Decision Or Authorization Date

17-12-2024

Processing Time Days

245

Number Of Sites

3

Number Of Participants

11

Primary sponsor

Full Name

Biomarin Pharmaceutical Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Development Solutions LLC

Responsibilities

Immunogenicity

Name

Parexel International (IRL) Limited

Responsibilities

codes: 1,12

Name

PPD International Holdings LLC

Responsibilities

Clinical chemistry and haematology

Third parties

• {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"Immunogenicity","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Greenwood Genetic Center Inc.","duties_or_roles":"Genotyping","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"codes: 1,12","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Alturas Analytics Inc.","duties_or_roles":"Primary/ surrogate endpoint test","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Eurofins Viracor Biopharma Services Inc.","duties_or_roles":"Immunogenicity","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Clinical chemistry and haematology","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"Analytical Chemistry","organisation_type":"Laboratory/Research/Testing facility"}