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.
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2018-000648-25-FR (EUCTR) | 05/07/2019 | 15/10/2018 | A 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 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 76 | Phase 3 | France;Spain;Turkey;Austria;Russian Federation;Netherlands;Germany;Italy | |||
| 2 | EUCTR2018-000648-25-DE (EUCTR) | 28/06/2019 | 30/08/2018 | A 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 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 85 | Phase 3 | France;Spain;Turkey;Austria;Netherlands;Germany;Italy | |||
| 3 | JPRN-JapicCTI-194642 | 13/6/2019 | 25/02/2019 | A Phase 3 Study to Assess the Safety and Efficacy of BMN 165 in Japanese Adults With Phenylketonuria | 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 | Phenylketonuria | Intervention 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. | NULL | complete | 18 | 70 | BOTH | 10 | Phase 3 | Japan |
| 4 | NCT01889862 (ClinicalTrials.gov) | July 29, 2013 | 18/6/2013 | Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU | A 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: Placebo | BioMarin Pharmaceutical | NULL | Completed | 18 Years | 70 Years | All | 215 | Phase 3 | United States |
| 5 | NCT01819727 (ClinicalTrials.gov) | May 2013 | 18/3/2013 | An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 | A 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 165 | Phenylketonuria | Drug: BMN 165 | BioMarin Pharmaceutical | NULL | Completed | 18 Years | 70 Years | All | 261 | Phase 3 | United States |
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT01560286 (ClinicalTrials.gov) | May 2012 | 5/3/2012 | A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks | A 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 Weeks | Phenylketonuria | Biological: BMN 165 (rAvPAL-PEG) | BioMarin Pharmaceutical | NULL | Completed | 16 Years | 70 Years | All | 24 | Phase 2 | United States |
| 7 | EUCTR2018-000648-25-NL (EUCTR) | 13/11/2018 | A 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 | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 85 | Phase 3 | France;Spain;Turkey;Austria;Germany;Netherlands;Italy |