240. Phenylketonuria Clinical trials / Disease details


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

  
4 trials found
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1NCT01395394
(ClinicalTrials.gov)
June 201123/6/2011Phenylketonuria, Oxidative Stress, and BH4The Ability of Kuvan® (Sapropterin Dihydrochloride) to Prevent Meal-induced Lipid Peroxidation and Endothelial Dysfunction in Patients With Phenylketonuria: a Pilot StudyPhenylketonuriaDrug: Kuvan;Other: Meal ChallengeEmory UniversityNULLTerminated10 Years45 YearsAll12Phase 2United States
2NCT01274026
(ClinicalTrials.gov)
January 201110/1/2011Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan NonrespondersEvaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression in PKU Nonresponders to Kuvan® (Sapropterin Dihydrochloride)Phenylketonuria;Behavior and Behavior Mechanisms;PAH Gene ExpressionDrug: sapropterin dihydrochlorideTulane University School of MedicineBioMarin PharmaceuticalCompleted2 YearsN/AAll21United States
3EUCTR2021-003777-63-NL
(EUCTR)
24/11/2021BH4 (Kuvan©) treatment in Phenylketonuria - comparing different practices of dosing regimensTetrahydrobiopterin (BH4) treatment in Phenylketonuria - comparing different practices of dosing regimen - BH4 dose Phenylketonuria (PKU; OMIM 261600);Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Trade Name: Kuvan® (sapropterin dihydrochloride)
Product Name: Kuvan® (sapropterin dihydrochloride)
Product Code: A16AX07
University Medical Center GroningenNULLNAFemale: yes
Male: yes
28Phase 4Netherlands
4EUCTR2009-012978-12-NO
(EUCTR)
02/06/2009A Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in NorwayA Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in Norway Hyperphenylalaninemia (HPA) in adults and children >=16 years of age with phenylketonuri (PKU) (classic or mild PKU, or mild hyperphenylalaninemia (HPA)).
MedDRA version: 9.1;Level: LLT;Classification code 10034872;Term: Phenylketonuria
MedDRA version: 9.1;Classification code 10034873;Term: Phenylketonuria (PKU)
MedDRA version: 9.1;Level: PT;Classification code 10034871;Term: Phenylalaninemia
Trade Name: Kuvan® (sapropterin dihydrochloride)
INN or Proposed INN: sapropterin
Merck Serono, an affiliate of E. Merck ABNULLNot RecruitingFemale: yes
Male: yes
Phase 4Norway