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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
11 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-FR05/07/201915/10/2018A 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
76 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noFrance;Spain;Turkey;Austria;Russian Federation;Netherlands;Germany;Italy
2EUCTR2018-000648-25-DE28/06/201930/08/2018A 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
85 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noFrance;Spain;Turkey;Austria;Netherlands;Germany;Italy
3JPRN-JapicCTI-19464213/6/201925/02/2019A 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
4NCT03792451January 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
5NCT03694353September 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
6NCT01560286May 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
7NCT01212744March 201129/9/2010Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With PhenylketonuriaPhenylketonuriaDrug: rAvPAL-PEGBioMarin PharmaceuticalNULLCompleted16 Years70 YearsAll16Phase 2United States
8NCT00924703October 8, 200918/6/2009Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects With PKUPhenylketonuriaDrug: rAvPAL-PEGBioMarin PharmaceuticalNULLCompleted16 Years55 YearsAll68Phase 2United States
9NCT00925054September 200917/6/2009Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKUPhase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKUPhenylketonuriaDrug: rAvPAL-PEG 0.001 mg/kg;Drug: rAvPAL-PEG 0.003 mg/kg;Drug: rAvPAL-PEG 0.01 mg/kg;Drug: rAvPAL-PEG 0.03 mg/kg;Drug: rAvPAL-PEG 0.1 mg/kgBioMarin PharmaceuticalNULLCompleted16 Years55 YearsAll40Phase 2United States
10NCT00634660May 20086/3/2008Safety and Tolerability Study of rAvPAL-PEG to Treat PhenylketonuriaA Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single, Subcutaneous Doses of rAvPAL-PEG in Subjects With PhenylketonuriaPhenylketonuriaDrug: rAvPAL-PEGBioMarin PharmaceuticalNULLCompleted16 Years50 YearsAll25Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2018-000648-25-NL13/11/2018A 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
85 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): no Therapeut1c conf1rmatory - (Phase 3): yes Therapeut1c use (Phase 4): noFrance;Spain;Turkey;Austria;Germany;Netherlands;Italy