240. Phenylketonuria Clinical trials / Disease details

Clinical trials : 138 / Drugs : 106 - (DrugBank : 11) / Drug target gene : 1 - Drug target pathways : 5

2 trials found

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	NCT02677870 (ClinicalTrials.gov)	January 2018	26/1/2016	The Effectiveness of Kuvan in Amish PKU Patients	The Effectiveness of High-Dose Synthetic BH4 (Saproterin Dihydrochloride or Kuvan) in Amish PKU Patients	Phenylketonuria	Drug: saproterin dihydrochloride;Other: Diet treatment	University Hospitals Cleveland Medical Center	BioMarin Pharmaceutical	Recruiting	2 Years	60 Years	All	25	Phase 4	United States
2	EUCTR2005-003778-13-DE (EUCTR)	13/07/2006	14/06/2006	A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency	A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 Deficiency	Phenylketonuria (PKU) MedDRA version: 8.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria	Product Name: Phenoptin Product Code: T1401 INN or Proposed INN: Sapropterin Other descriptive name: saproterin dihydrochloride	BioMarin Pharmaceutical Inc	NULL	Not Recruiting			Female: yes Male: yes	15	Phase 2	Germany