240. Phenylketonuria
125 clinical trials, 95 drugs (DrugBank: 11 drugs), 1 drug target gene, 5 drug target pathways
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 | 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 | Recruiting | 18 Years | 65 Years | All | 20 | United States | |
| 5 | NCT03694353 (ClinicalTrials.gov) | September 13, 2018 | 4/9/2018 | Study to Evaluate the 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 | Active, not recruiting | 18 Years | 70 Years | All | 37 | 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 | NCT01212744 (ClinicalTrials.gov) | March 2011 | 29/9/2010 | Safety, 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 Phenylketonuria | Phenylketonuria | Drug: rAvPAL-PEG | BioMarin Pharmaceutical | NULL | Completed | 16 Years | 70 Years | All | 16 | Phase 2 | United States |
| 8 | NCT00924703 (ClinicalTrials.gov) | October 8, 2009 | 18/6/2009 | Long-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 PKU | Phenylketonuria | Drug: rAvPAL-PEG | BioMarin Pharmaceutical | NULL | Completed | 16 Years | 55 Years | All | 68 | Phase 2 | United States |
| 9 | NCT00925054 (ClinicalTrials.gov) | September 2009 | 17/6/2009 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU | Phenylketonuria | Drug: 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/kg | BioMarin Pharmaceutical | NULL | Completed | 16 Years | 55 Years | All | 40 | Phase 2 | United States |
| 10 | NCT00634660 (ClinicalTrials.gov) | May 2008 | 6/3/2008 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | A Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single, Subcutaneous Doses of rAvPAL-PEG in Subjects With Phenylketonuria | Phenylketonuria | Drug: rAvPAL-PEG | BioMarin Pharmaceutical | NULL | Completed | 16 Years | 50 Years | All | 25 | Phase 1 | 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 |
| 11 | 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 |