PYRIMETHAMINE ( DrugBank: Pyrimethamine )

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4 diseases

ID	Disease name (Link within this page)	Number of trials
2	Amyotrophic lateral sclerosis	2
4	Primary lateral sclerosis	1
19	Lysosomal storage disease	2
65	Primary immunodeficiency	2

2. Amyotrophic lateral sclerosis

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Clinical trials : 645 /  Drugs : 589 - (DrugBank : 163) / Drug target genes : 150 - Drug target pathways : 225

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	EUCTR2011-004798-99-DE (EUCTR)	27/02/2013	27/12/2012	Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS)	Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS) - Pyrimethamine In Familial ALS	Familial amyotrophic lateral sclerosis (FALS) MedDRA version: 14.1;Level: LLT;Classification code 10036704;Term: Primary lateral sclerosis;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: Daraprim INN or Proposed INN: PYRIMETHAMINE	Weill Medical College of Cornell University	NULL	Not Recruiting			Female: yes Male: yes	40		United States;Germany;Italy
2	NCT01083667 (ClinicalTrials.gov)	November 2009	17/12/2009	SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)	Phase I/II Study of SOD1 Inhibition by Pyrimethamine in Familial ALS	Familial Amyotrophic Lateral Sclerosis	Drug: Pyrimethamine	Weill Medical College of Cornell University	Muscular Dystrophy Association	Completed	18 Years	N/A	All	32	Phase 1/Phase 2	United States;Germany;Italy;Sweden

4. Primary lateral sclerosis

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Clinical trials : 5 /  Drugs : 13 - (DrugBank : 6) / Drug target genes : 13 - Drug target pathways : 25

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	EUCTR2011-004798-99-DE (EUCTR)	27/02/2013	27/12/2012	Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS)	Safety and Efficacy of SOD1 Inhibition By Pyrimethamine in Familial (ALS) - Pyrimethamine In Familial ALS	Familial amyotrophic lateral sclerosis (FALS) MedDRA version: 14.1;Level: LLT;Classification code 10036704;Term: Primary lateral sclerosis;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Trade Name: Daraprim INN or Proposed INN: PYRIMETHAMINE	Weill Medical College of Cornell University	NULL	Not Recruiting			Female: yes Male: yes	40		United States;Germany;Italy

19. Lysosomal storage disease

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Clinical trials : 899 /  Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

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	NCT01102686 (ClinicalTrials.gov)	August 2009	16/10/2009	Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)	Proposed Investigator-Initiated Clinical Trial of Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)	Gangliosidoses, GM2;Sandhoff Disease;Tay-Sachs Disease	Drug: Pyrimethamine;Drug: Leucovorin	The Hospital for Sick Children	NULL	Completed	17 Years	N/A	Both	20	Phase 1/Phase 2	Canada
2	NCT00679744 (ClinicalTrials.gov)	May 2008	15/5/2008	A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis	A Dose-Escalated, Double-Blind, Placebo-Controlled, Randomized Phase I Clinical Trial of Pyrimethamine in Patients Affected With Chronic GM2 Gangliosidosis (Tay-Sachs or Sandhoff Variants)	G(M2) Ganglioside;Tay-Sachs Disease Ganglioside;Sandhoff Disease Ganglioside	Drug: Pyrimethamine	Exsar Corporation	University Hospital Case Medical Center;New York University School of Medicine;The Hospital for Sick Children	Withdrawn	18 Years	N/A	Both	0	Phase 1	United States;Canada

65. Primary immunodeficiency

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Clinical trials : 500 /  Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217

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	NCT00065390 (ClinicalTrials.gov)	July 2003	21/7/2003	Pyrimethamine to Treat Autoimmune Lymphoproliferative Syndrome	Pilot (Phase I-II) Study of Pyrimethamine (Daraprim) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS)	Autoimmune Disease;Lymphatic Disease;Lymphoproliferative Disorder	Drug: Pyrimethamine	National Institute of Allergy and Infectious Diseases (NIAID)	NULL	Completed	N/A	N/A	Both	8	Phase 1	United States
2	NCT00013689 (ClinicalTrials.gov)	March 2001	28/3/2001	Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative Syndrome	Pilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS)	Autoimmune Disease;Lymphoproliferative Disorder	Drug: Fansidar (pyrimethamine and sulfadoxine)	National Institute of Allergy and Infectious Diseases (NIAID)	NULL	Completed	N/A	N/A	Both	8	Phase 1	United States

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