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

臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5

74 trials found

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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	NCT04014712 (ClinicalTrials.gov)	August 2019	3/7/2019	O2 Transport and Utilization in Health and Lung Disease	Role of Nitric Oxide Coupling in Muscle Dysfunction With COPD	COPD;Tetrahydrobiopterin Deficiency;Oxidative Stress	Drug: Tetrahydrobiopterin;Drug: Placebo oral tablet	University of Massachusetts, Amherst	NULL	Not yet recruiting	18 Years	85 Years	All	30	Phase 1	United States
2	NCT03820804 (ClinicalTrials.gov)	January 7, 2019	19/10/2018	Nutritional Status in Phenylketonuria	Trends in Nutritional Status of Patients With Phenylketonuria	Phenylketonurias	Other: Diet;Drug: Sapropterin	Universidade do Porto	Centro Hospitalar do Porto;BioMarin Pharmaceutical	Completed	3 Years	N/A	All	94		Portugal
3	NCT03519711 (ClinicalTrials.gov)	June 24, 2018	11/4/2018	A Study of CNSA-001 in Primary Tetrahydrobiopterin(BH4) Deficient Participants With Hyperphenylalani ...	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
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	EUCTR2010-024311-13-NL (EUCTR)	13/05/2014	27/01/2014	A clinical study, carried out in different centers and using a placebo, to assess the effect of a me ...	A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ...	Phenylketonuria MedDRA version: 16.1;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ...	Trade Name: Kuvan ® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE ...	Merck KGaA	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 2	France;Spain;Belgium;Germany;Netherlands;Italy;Switzerland
6	EUCTR2010-021343-41-NL (EUCTR)	21/03/2014	30/11/2011	Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterin	Effect of sapropterinon variations of blood phenylalanine and tyrosine over 24 hours and from day to ...	Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene ...	Trade Name: Kuvan	University Medical Center Groningen	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 4	Netherlands
7	NCT01977820 (ClinicalTrials.gov)	February 2014	31/10/2013	Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria	A Multicenter, Double-Blind, Placebo-Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ...	Phenylketonuria	Drug: Sapropterin;Drug: Placebo	BioMarin Pharmaceutical	NULL	Terminated	18 Years	29 Years	All	2	Phase 2	Italy;Switzerland;Belgium;Germany;Netherlands;Spain
8	EUCTR2010-024311-13-DE (EUCTR)	02/01/2014	23/07/2013	A clinical study, carried out in different centers and using a placebo, to assess the effect of a me ...	A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ...	Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ...	Trade Name: Kuvan ® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE ...	Merck KGaA	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 2	France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland
9	EUCTR2010-024311-13-BE (EUCTR)	23/12/2013	25/09/2013	A clinical study, carried out in different centers and using a placebo, to assess the effect of a me ...	A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ...	Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ...	Trade Name: Kuvan ® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE ...	Merck KGaA	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 2	France;Spain;Belgium;Netherlands;Germany;Italy;Switzerland
10	EUCTR2010-024311-13-ES (EUCTR)	21/11/2013	01/10/2013	A clinical study, carried out in different centers and using a placebo, to assess the effect of a me ...	A Multicenter, Double-Blind, Placebo Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing t ...	Phenylketonuria MedDRA version: 16.0;Level: LLT;Classification code 10034873;Term: Phenylketonuria ( ...	Trade Name: Kuvan ® INN or Proposed INN: Sapropterin Other descriptive name: SAPROPTERIN DIHYDROCHLORIDE ...	Merck KGaA	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 2	France;Belgium;Spain;Netherlands;Germany;Italy;Switzerland

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