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Pegvaliase    (DrugBank: Pegvaliase)

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6

240. フェニルケトン尿症　［臨床試験数：125，薬物数：95（DrugBank：11），標的遺伝子数：1，標的パスウェイ数：5］
Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
6 / 125 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		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]		BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes		76Phase 3France;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]		BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes		85Phase 3France;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
4NCT03792451
(ClinicalTrials.gov)		January 17, 201931/12/2018Nutrition Status of Adults Treated With PegvaliaseNutrition Status of Adults With Phenylketonuria Treated With PegvaliasePhenylketonuriasDrug: PegvaliaseBoston Children's HospitalBioMarin PharmaceuticalRecruiting18 Years65 YearsAll20United States
5NCT03694353
(ClinicalTrials.gov)		September 13, 20184/9/2018Study to Evaluate the Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKUAn Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With PhenylketonuriaPhenylketonuria (PKU)Drug: PegvaliaseBioMarin PharmaceuticalNULLActive, not recruiting18 Years70 YearsAll37Phase 3United States
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
6EUCTR2018-000648-25-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]		BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes		85Phase 3France;Spain;Turkey;Austria;Germany;Netherlands;Italy

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