A Phase 3, Open-Label, Multi-Center Study to Assess the Safety and Efficacy of BMN 165 in Japanese Subjects 18 Years of Age and Older With Phenylketonuria
A Phase 3 Study to Assess the Safety and Efficacy of BMN 165 in Japanese Adults With Phenylketonuria
CMIC Co., Ltd.
Hamamatsucho Bldg., 1-1-1 Shibaura, Minatoku, Tokyo
ClinicalTrialInformation@cmic.co.jp
CMIC Co., Ltd.
Hamamatsucho Bldg., 1-1-1 Shibaura, Minatoku, Tokyo
+81-3-6779-8000
ClinicalTrialInformation@cmic.co.jp
completed
June. 13, 2019
10
Interventional
Screening :4 weeks in duration
Part 1 : Phase 3, Open-Label, Multi-Center Study which is 52 weeks consisting of Induction/Titration period and Maintenance period.
Part 2 : Long-term Extension period (up to 156 weeks).
treatment purpose
3
(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.
(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.
18age old over
70age old under
Both
Phenylketonuria
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 Investigational material : BMN 165 will be administered SC at dose levels of 2.5 to 60 mg. The minimum dose is a single weekly dose of 2.5 mg (for a total weekly dose of 2.5 mg). The maximum allowable daily dose is 40 mg/day (for a maximum weekly dose of 280 mg) after a minimum of 24 weeks on 20 mg/day during Part 1. Subjects may increase dose up to 60 mg/day in Part 2 (for a maximum weekly dose of 420 mg) after a minimum of 16 weeks on 40 mg/day.
control material(s)
Generic name etc : -
INN of investigational material : -
Therapeutic category code : --- Other
Dosage and Administration for Investigational material : -
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.
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.
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.
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Osaka City University Hospital Institutional Review Board
1-5-7 Asahi-machi, Abeno-ku, Osaka, Japan
+81-6-6645-2121
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approved
Jan. 31, 2019
The Central Institutional Review Board of the Fujita Health University Hospitals
1-98 Dengakugakubo, Kutsukake-cho, Toyoake-shi, Aichi, Japan
+81-562-93-2111
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approved
Jan. 31, 2019
Nihon University Hospital's Joint Institutional Review Board
