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

22 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	NCT03910621 (ClinicalTrials.gov)	April 2, 2020	8/3/2019	Safety and Efficacy of Miglustat in Chinese NPC Patients	A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat( ...	Niemann-Pick Disease, Type C	Drug: Miglustat	Actelion	NULL	Completed	4 Years	N/A	All	17	Phase 4	China
2	NCT03822013 (ClinicalTrials.gov)	January 14, 2019	22/1/2019	Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)	Survey of MiglustatTherapeutic Effects on Neurological and Systemic Symptoms of Infantile Type of Sa ...	GM2 Gangliosidosis;Supportive Care	Drug: Miglustat	Tehran University of Medical Sciences	Mashhad University of Medical Sciences;Kashan University of Medical Sciences	Recruiting	6 Months	24 Months	All	30	Phase 3	Iran, Islamic Republic of
3	NCT02030015 (ClinicalTrials.gov)	December 22, 2015	17/12/2013	Synergistic Enteral Regimen for Treatment of the Gangliosidoses	Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G)	GM1 Gangliosidoses;GM2 Gangliosidoses;Tay-Sachs Disease;Sandhoff Disease	Drug: miglustat;Other: Ketogenic Diet	University of Minnesota	Rare Diseases Clinical Research Network;National Center for Advancing Translational Science (NCATS); ...	Recruiting	N/A	204 Months	All	30	Phase 4	United States
4	EUCTR2014-005194-37-DE (EUCTR)	19/10/2015	28/04/2015	A non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the in ...	A prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order ...	Niemann-Pick disease - type C MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niem ...	Trade Name: MIglustat Product Name: Zavesca Product Code: A16AX06 INN or Proposed INN: MIGLUSTAT	Orphazyme ApS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 1	Spain;Germany;Switzerland
5	EUCTR2014-005194-37-ES (EUCTR)	13/07/2015	08/05/2015	A non-therapeutic study in patients with Niemann-Pick disease type C in order to characterise the in ...	A prospective non-therapeutic study in patients diagnosed with Niemann-Pick disease type C in order ...	Niemann-Pick disease - type C MedDRA version: 18.0;Level: PT;Classification code 10029403;Term: Niem ...	Trade Name: MIglustat Product Name: Zavesca Product Code: A16AX06 INN or Proposed INN: MIGLUSTAT	Orphazyme ApS	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 1	Spain;Germany;Switzerland
6	NCT02520934 (ClinicalTrials.gov)	July 2015	6/8/2015	Miglustat on Gaucher Disease Type IIIB	Evaluation of Combination Therapy With Miglustatand Enzyme Replacement Therapy on Gaucher Disease Ty ...	Gaucher Disease	Drug: Miglustat;Drug: ERT	National Taiwan University Hospital	Actelion	Active, not recruiting	6 Years	N/A	All	19	N/A	Taiwan
7	EUCTR2007-005516-61-IT (EUCTR)	06/02/2009	23/03/2009	Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high ...	Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high ...	GAUCHER disease MedDRA version: 9.1;Level: LLT;Classification code 10018048;Term: Gaucher's disease	Trade Name: CEREZYME INN or Proposed INN: Imiglucerase Trade Name: ZAVESCA INN or Proposed INN: Miglust ...	ISTITUTO GIANNINA GASLINI	NULL	Not Recruiting			Female: yes Male: yes			Italy
8	EUCTR2007-007164-20-ES (EUCTR)	20/05/2008	21/02/2008	Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes co ...	Uso racional de los tratamientos por inhibición de sustrato y enzimático sustitutivo en pacientes co ...	Tratamiento de la enfermedad de Gaucher MedDRA version: 9.1;Level: LLT;Classification code 10018048; ...	Trade Name: CEREZYME INN or Proposed INN: IMIGLUCERASA Trade Name: MIGLUSTAT (ZAVESCA) INN or Proposed I ...	INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD	NULL	Not Recruiting			Female: yes Male: yes			Spain
9	NCT01760564 (ClinicalTrials.gov)	January 2008	2/1/2013	Application of Miglustat in Patients With Niemann-Pick Type C	Application of Miglustat in Patients With Niemann-Pick Type C	Niemann-Pick Disease Type C	Drug: Miglustat	National Taiwan University Hospital	NULL	Completed	N/A	N/A	Both	5	Phase 3	Taiwan
10	EUCTR2005-001651-37-HU (EUCTR)	10/12/2007	04/10/2007	Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and toler ...	Open-label, non-comparative, multi-center study to evaluate the long-term efficacy, safety and toler ...	Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal reces ...	Trade Name: Zavesca Product Name: Miglustat Product Code: OGT918 INN or Proposed INN: miglustat	Actelion Pharmaceuticals Ltd	NULL	Not Recruiting			Female: yes Male: yes	50		Hungary;Czech Republic;Germany;United Kingdom;Spain;Italy

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