19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details

臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

16 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	NCT04246060 (ClinicalTrials.gov)	July 31, 2020	27/1/2020	Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients	Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectivenes ...	Nephropathic Cystinosis	Drug: Cysteamine Bitartrate	Chiesi SA/NV	NULL	Enrolling by invitation	N/A	N/A	All	31		Belgium
2	EUCTR2012-002773-64-IT (EUCTR)	12/02/2014	21/11/2013	A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ...	A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ...	Cystinosis MedDRA version: 16.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ...	Product Name: Cysteamine bitartrate(INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed ...	Raptor Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	France;United States;Belgium;Netherlands;Germany;United Kingdom;Italy
3	EUCTR2012-002773-64-GB (EUCTR)	22/01/2014	26/02/2013	Study comparing the effectiveness of cysteamine bitartratedelayed release capsules (RP103) to Cystag ...	A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ...	Cystinosis MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ...	Trade Name: PROCYSBI Product Name: Cysteamine bitartrate(INN: mercaptamine bitartrate ) Product Code: R ...	Horizon Pharma USA, Inc.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	France;United States;Belgium;Netherlands;Germany;Italy;United Kingdom
4	EUCTR2012-002773-64-BE (EUCTR)	21/01/2014	19/04/2013	A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine BitartrateDelayed-rele ...	A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine BitartrateDelayed-rele ...	Cystinosis MedDRA version: 14.1;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ...	Product Name: Cysteamine bitartrate(INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed ...	Raptor Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	United States;France;Belgium;Netherlands;Germany;United Kingdom;Italy
5	EUCTR2012-002773-64-NL (EUCTR)	15/08/2013	01/11/2013	Study comparing the effectiveness of cysteamine bitartratedelayed-release capsules (RP103) in Patien ...	A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine BitartrateDelayed-rele ...	Cystinosis MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ...	Trade Name: PROCYSBI Product Name: Cysteamine bitartrate(INN: mercaptamine bitartrate) Product Code: RP ...	Horizon Pharma USA, Inc.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	France;United States;Belgium;Germany;Netherlands;Italy;United Kingdom
6	EUCTR2012-002773-64-FR (EUCTR)	03/06/2013	28/09/2015	A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ...	A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-rele ...	Cystinosis MedDRA version: 18.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ...	Product Name: Cysteamine bitartrate(INN: mercaptamine bitartrate ) Product Code: RP103 INN or Proposed ...	Raptor Therapeutics Inc.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	United States;France;Belgium;Netherlands;Italy;United Kingdom
7	NCT01733316 (ClinicalTrials.gov)	January 31, 2013	16/11/2012	Open-Label, Safety and Superior Effectiveness Study of Cysteamine BitartrateDelayed-Release Capsules ...	A Long-Term, Open-Label, Safety, Tolerability and Superior Effectiveness Study of Cysteamine Bitartr ...	Cystinosis	Drug: RP103;Drug: Cystagon ®	Horizon Pharma USA, Inc.	NULL	Completed	12 Years	N/A	All	41	Phase 3	United States;Belgium;France;Italy;Netherlands;United Kingdom
8	NCT01744782 (ClinicalTrials.gov)	December 20, 2012	5/12/2012	Safety/Effectiveness Study of Cysteamine BitartrateDelayed-release Capsules (RP103) in Cysteamine Tr ...	An Open-Label, Safety and Effectiveness Study of Cysteamine BitartrateDelayed-release Capsules (RP10 ...	Cystinosis	Drug: RP103	Horizon Pharma USA, Inc.	NULL	Completed	N/A	6 Years	All	17	Phase 3	United States;Brazil
9	NCT01432561 (ClinicalTrials.gov)	September 2011	9/9/2011	Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the ...	Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults	Cystinosis;Nephropathic Cystinosis	Drug: Cysteamine bitartrate	University of California, San Diego	Raptor Pharmaceuticals Corp.	Completed	18 Years	N/A	All	8	N/A	United States
10	EUCTR2010-018365-34-NL (EUCTR)	14/12/2010	06/07/2010	A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine BitartrateDelayed-release Capsules ...	A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine BitartrateDelayed-release Capsules ...	Cystinosis MedDRA version: 20.0;Level: PT;Classification code 10011777;Term: Cystinosis;System Organ ...	Trade Name: PROCYSBI Product Name: Cysteamine bitartrate(INN: mercaptamine bitartrate ) Product Code: R ...	Horizon Pharma USA, Inc.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	France;United States;Netherlands

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