BMN 165 ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
240フェニルケトン尿症7

240. フェニルケトン尿症


臨床試験数 : 143 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1EUCTR2018-000648-25-FR
(EUCTR)
05/07/201915/10/2018A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current ManagementA Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study Phenylketonuria (PKU)
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
76 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Spain;Turkey;Austria;Russian Federation;Netherlands;Germany;Italy
2EUCTR2018-000648-25-DE
(EUCTR)
28/06/201930/08/2018A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current ManagementA Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study Phenylketonuria (PKU)
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
85 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Spain;Turkey;Austria;Netherlands;Germany;Italy
3JPRN-JapicCTI-194642
13/6/201925/02/2019A Phase 3 Study to Assess the Safety and Efficacy of BMN 165 in Japanese Adults With PhenylketonuriaA 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 PhenylketonuriaIntervention name : BMN 165
INN of the intervention : pegvaliase
Dosage And administration of the intervention : 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 intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -
BioMarin Pharmaceutical Inc./CMIC Co., Ltd.NULLcomplete1870BOTH10Phase 3Japan
4NCT01889862
(ClinicalTrials.gov)
July 29, 201318/6/2013Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKUA Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)Phenylketonuria (PKU)Drug: BMN165 20mg/day;Drug: BMN165 40mg/day;Drug: PlaceboBioMarin PharmaceuticalNULLCompleted18 Years70 YearsAll215Phase 3United States
5NCT01819727
(ClinicalTrials.gov)
May 201318/3/2013An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety & Tolerability of an Induction, Titration, and Maintenance Dose Regimen of BMN 165 Self Administered by Adults With PKU Not Previously Treated With BMN 165PhenylketonuriaDrug: BMN 165BioMarin PharmaceuticalNULLCompleted18 Years70 YearsAll261Phase 3United States
6NCT01560286
(ClinicalTrials.gov)
May 20125/3/2012A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 WeeksA Phase II, Multi-center, Open-label, Dose-finding Study to Evaluate Safety, Efficacy and Tolerability of Subcutaneously (SC) Administered rAvPAL-PEG in Patients With PKU for 24 WeeksPhenylketonuriaBiological: BMN 165 (rAvPAL-PEG)BioMarin PharmaceuticalNULLCompleted16 Years70 YearsAll24Phase 2United States
7EUCTR2018-000648-25-NL
(EUCTR)
13/11/2018A Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current ManagementA Phase 3b, Open-Label, Single Arm, Multi-Centre Study to Assess the Safety and Efficacy of Pegvaliase (BMN 165) Treatment in adults With Phenylketonuria Not Controlled With Current Management - OPTIC Study Phenylketonuria (PKU)
MedDRA version: 20.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria (PKU);System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
Product Name: Pegvaliase
INN or Proposed INN: PEGVALIASE
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
85 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Spain;Turkey;Austria;Germany;Netherlands;Italy