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 | 9 |
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. | 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 | NCT05579548 (ClinicalTrials.gov) | October 4, 2022 | 11/10/2022 | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding | Phenylketonuria, Maternal | Drug: Pegvaliase | BioMarin Pharmaceutical | Syneos Health | Recruiting | N/A | N/A | Female | 50 | United States | |
| 2 | NCT05270837 (ClinicalTrials.gov) | June 17, 2022 | 1/2/2022 | Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria | A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design | Phenylketonuria (PKU) | Drug: Pegvaliase;Other: Diet Only | BioMarin Pharmaceutical | NULL | Recruiting | 12 Years | 17 Years | All | 54 | Phase 3 | United States;Germany |
| 3 | NCT05356377 (ClinicalTrials.gov) | May 25, 2022 | 27/4/2022 | Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU | A Preliminary Study of the Neurological and Neuropsychological Effects of Palynziq-Related Changes in Phenylalanine in Individuals With Phenylketonuria (PKU) | Phenylketonurias | Drug: Pegvaliase-Pqpz | University of Missouri-Columbia | BioMarin Pharmaceutical | Enrolling by invitation | 18 Years | 55 Years | All | 13 | United States | |
| 4 | 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] | 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. | NULL | Not 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): no | France;Spain;Turkey;Austria;Russian Federation;Netherlands;Germany;Italy | ||
| 5 | 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] | 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. | NULL | Not 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): no | France;Spain;Turkey;Austria;Netherlands;Germany;Italy | ||
| 6 | 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 |
| 7 | NCT03792451 (ClinicalTrials.gov) | January 17, 2019 | 31/12/2018 | Nutrition Status of Adults Treated With Pegvaliase | Nutrition Status of Adults With Phenylketonuria Treated With Pegvaliase | Phenylketonurias | Drug: Pegvaliase | Boston Children's Hospital | BioMarin Pharmaceutical | Completed | 18 Years | 65 Years | All | 18 | United States | |
| 8 | NCT03694353 (ClinicalTrials.gov) | September 13, 2018 | 4/9/2018 | Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU | An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria | Phenylketonuria (PKU) | Drug: Pegvaliase | BioMarin Pharmaceutical | NULL | Completed | 18 Years | 70 Years | All | 37 | Phase 3 | United States |
| 9 | 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] | 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. | NULL | Not 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): no | France;Spain;Turkey;Austria;Germany;Netherlands;Italy |