240. Phenylketonuria Clinical trials / Disease details
Clinical trials : 138 / Drugs : 106 - (DrugBank : 11) / Drug target gene : 1 - Drug target pathways : 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 | 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 |
| 2 | 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 |
| 3 | 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 |
| 4 | 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 |
| 5 | 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 |