240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details

臨床試験数 : 138 / 薬物数 : 106 - (DrugBank : 11) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5

17 trials found

diseases per page:

Showing 1 to 10 of 17 diseases

Filter by

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	NCT04227080 (ClinicalTrials.gov)	June 2020	10/1/2020	BH4 Responsiveness in PAH Deficiency PKU Patients	To Evaluate BH4Responsiveness in PAH Deficiency PKU Patients Who Failed to Achieve 30% Blood Phe Red ...	Pku Phenylketonuria;PAH Deficiency	Drug: BH4	Taipei Veterans General Hospital, Taiwan	BioMarin Pharmaceutical	Not yet recruiting	N/A	N/A	All	40	Phase 4	NULL
2	NCT03519711 (ClinicalTrials.gov)	June 24, 2018	11/4/2018	A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalan ...	A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate th ...	BH4 Deficiency;Hyperphenylalaninemia	Drug: CNSA-001	PTC Therapeutics	NULL	Completed	12 Months	N/A	All	6	Phase 1/Phase 2	United States;Germany
3	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 Patie ...	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
4	NCT03864029 (ClinicalTrials.gov)	October 10, 2017	22/2/2019	Retrospective Observational Safety Effectiveness With Kuvan in hpA	An Observational Study Research to Collect the Effectiveness and Safety Data of KUVAN® Retrospective ...	Tetrahydrobiopterin Deficiency	Drug: KUVAN	BioMarin Pharmaceutical	Quintiles, Inc.	Completed	N/A	N/A	All	26		China
5	NCT01395394 (ClinicalTrials.gov)	June 2011	23/6/2011	Phenylketonuria, Oxidative Stress, and BH4	The Ability of Kuvan® (Sapropterin Dihydrochloride) to Prevent Meal-induced Lipid Peroxidation and E ...	Phenylketonuria	Drug: Kuvan;Other: Meal Challenge	Emory University	NULL	Terminated	10 Years	45 Years	All	12	Phase 2	United States
6	EUCTR2010-019767-11-AT (EUCTR)	08/09/2010	10/08/2010	Ö-PKU 1 – Evaluation of a Test for the identication of BH4 responsive PKU patients - Ö-PKU1	Ö-PKU 1 – Evaluation of a Test for the identication of BH4 responsive PKU patients - Ö-PKU1	Hyperphenylalaninaemia (HPA) in adult and paediatric patients of 4 years of age and over with phenyl ...	Trade Name: Kuvan	Graz Medical University	NULL	Not Recruiting			Female: yes Male: yes			Austria
7	NCT00986973 (ClinicalTrials.gov)	March 2010	28/9/2009	Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria ...	A Pilot Study of FDG PET Findings in Patients With Phenylketonuria Before and After BH4Supplementati ...	Phenylketonuria	Drug: Sapropterin	Children's Hospital of Philadelphia	NULL	Completed	18 Years	50 Years	All	6	N/A	United States
8	NCT00688844 (ClinicalTrials.gov)	October 2008	29/5/2008	Nutritional and Neurotransmitter Changes in PKU Subjects on BH4	Baseline Evaluation and Long-term Follow-up of Nutritional Status and Neurotransmitter Concentration ...	Phenylketonuria	Drug: KuvanTM Therapy	Emory University	BioMarin Pharmaceutical;Atlanta Clinical and Translational Science Institute	Completed	4 Years	N/A	All	58	N/A	United States
9	NCT00432822 (ClinicalTrials.gov)	February 2007	7/2/2007	Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Toler ...	Double-Blind, Placebo Controlled, Multicentre Study With an Open Label Extension to Evaluate the Eff ...	Phenylalanine Hydroxylase Deficiencies	Drug: tetrahydrobiopterin (BH4)	Orphanetics Pharma Entwicklungs GmbH	NULL	Terminated	N/A	18 Years	Both	50	Phase 2/Phase 3	NULL
10	NCT00355264 (ClinicalTrials.gov)	August 2006	19/7/2006	Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency	Phase 2, Multicenter, Open Label Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to Pr ...	Tetrahydrobiopterin Deficiencies;Hyperphenylalaninemia, Non-Phenylketonuric	Drug: Phenoptin	BioMarin Pharmaceutical	NULL	Completed	N/A	N/A	All	12	Phase 2	United States;Germany

First Previous1 2Next Last