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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02677870
(ClinicalTrials.gov)
January 201826/1/2016The Effectiveness of Kuvan in Amish PKU PatientsThe Effectiveness of High-Dose Synthetic BH4 (Saproterin Dihydrochloride or Kuvan) in Amish PKU PatientsPhenylketonuriaDrug: saproterin dihydrochloride;Other: Diet treatmentUniversity Hospitals Cleveland Medical CenterBioMarin PharmaceuticalRecruiting2 Years60 YearsAll25Phase 4United States
2EUCTR2005-003778-13-DE
(EUCTR)
13/07/200614/06/2006A Phase 2, Multicenter, Open-label Study to Evaluate the Safety and Efficacy of Phenoptin in Subjects with Hyperphenylalaninemia Due to Primary BH4 DeficiencyA 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 IncNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Germany