Phenylketonuria
Conditions
Interventions
investigational material(s)
Generic name etc : BMN 165
INN of investigational material : pegvaliase
Therapeutic category code : 399 Agents affecting metabolism, n.e.c.
Dosage and Administration for In
Sponsors
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (Main items only) 1.Current diagnosis of PKU with 2 blood Phe concentrations > 600 micromole per liter during the Screening period (2 to 3 weeks between Phe assessments). 2.Average blood Phe concentration of > 600 micromole per liter over the past 6 months (per available data). 3.18 or more years of age and 70 or less years of age at the start of the Screening period (Day -28). 4.Has identified a competent adult 20 or more years of age who can observe the subject during study drug administration and for a minimum of 1 hour following administration for first 6 months of dosing, upon return to dosing after an AE, if dosing is increased, or per investigator determination. A home healthcare nurse may perform the study drug observations as acceptable by local regulatory and site requirements. 5.Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females are considered not to have childbearing potential if they have been in menopause for at least 2 years, have had a tubal ligation at least 1 year prior to screening, or have had a total hysterectomy. 6.If sexually active must agree to either not have sex during this study or 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
Exclusion criteria: (Main items only) 1. Previous treatment with BMN 165. 2. History of hypersensitivity to components of BMN 165. 3. Use of any investigational product or investigational medical device within 30 days prior to Screening (Day-28) or requirement for any investigational agent prior to completion of all scheduled study assessments. 4. Use of sapropterin (Biopten) treatment within 14 days prior to Day 1 (ie, first dose of BMN 165) or any other medication that is intended to treat PKU within 2 days prior to Day 1. 5. Use or planned use of any injectable drugs containing PEG (other than BMN 165), including medroxyprogesterone injection, within 3 months prior to Screening (Day-28) and during study participation. 6. Positive test for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, or hepatitis C antibody. 7. Pregnant or breastfeeding at Screening (Day -28) or planning to become pregnant (self or partner) or breastfeed at any time during the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy The primary objective of the study is to evaluate the efficacy and safety of BMN 165. The primary efficacy evaluations are observed blood Phe concentration,and change in blood Phe concentration and percentage change in blood Phe concentration from treatment naive baseline. Safety variables will be assessed for AEs, clinical laboratory tests, vital signs, physical examination, ECG and immunogenicity tests. | — |
Secondary
| Measure | Time frame |
|---|---|
| pharmacokinetics The secondary objective of the study is to characterize the PK of BMN 165. Through and intensive plasma BMN 165 concentrations will be summarized. | — |
Countries
Japan
Outcome results
None listed