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Pyrimethamine    (DrugBank: Pyrimethamine)

4 diseases
告示番号疾患名（ページ内リンク）臨床試験数
2筋萎縮性側索硬化症2
4原発性側索硬化症1
19ライソゾーム病2
65原発性免疫不全症候群2

2. 筋萎縮性側索硬化症　［臨床試験数：508，薬物数：530（DrugBank：146），標的遺伝子数：170，標的パスウェイ数：221］
Searched query = "Amyotrophic lateral sclerosis", "ALS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 508 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2011-004798-99-DE
(EUCTR)		27/02/201327/12/2012Safety 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 UniversityNULLNot RecruitingFemale: yes
Male: yes		40United States;Germany;Italy
2NCT01083667
(ClinicalTrials.gov)		November 200917/12/2009SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)Phase I/II Study of SOD1 Inhibition by Pyrimethamine in Familial ALSFamilial Amyotrophic Lateral SclerosisDrug: PyrimethamineWeill Medical College of Cornell UniversityMuscular Dystrophy AssociationCompleted18 YearsN/AAll32Phase 1;Phase 2United States;Germany;Italy;Sweden

4. 原発性側索硬化症　［臨床試験数：6，薬物数：16（DrugBank：8），標的遺伝子数：19，標的パスウェイ数：32］
Searched query = "Primary lateral sclerosis", "PLS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 6 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2011-004798-99-DE
(EUCTR)		27/02/201327/12/2012Safety 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 UniversityNULLNot RecruitingFemale: yes
Male: yes		40United States;Germany;Italy

19. ライソゾーム病　［臨床試験数：784，薬物数：673（DrugBank：101），標的遺伝子数：68，標的パスウェイ数：184］
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 784 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01102686
(ClinicalTrials.gov)		August 200916/10/2009Pyrimethamine 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 DiseaseDrug: Pyrimethamine;Drug: LeucovorinThe Hospital for Sick ChildrenNULLCompleted17 YearsN/ABoth20Phase 1;Phase 2Canada
2NCT00679744
(ClinicalTrials.gov)		May 200815/5/2008A Phase I Study of Pyrimethamine in Patients With GM2 GangliosidosisA 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 GangliosideDrug: PyrimethamineExsar CorporationUniversity Hospital Case Medical Center;New York University School of Medicine;The Hospital for Sick ChildrenWithdrawn18 YearsN/ABoth0Phase 1United States;Canada

65. 原発性免疫不全症候群　［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 413 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT00065390
(ClinicalTrials.gov)		July 200321/7/2003Pyrimethamine to Treat Autoimmune Lymphoproliferative SyndromePilot (Phase I-II) Study of Pyrimethamine (Daraprim) for the Treatment of the Autoimmune Lymphoproliferative Syndrome (ALPS)Autoimmune Disease;Lymphatic Disease;Lymphoproliferative DisorderDrug: PyrimethamineNational Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth8Phase 1United States
2NCT00013689
(ClinicalTrials.gov)		March 200128/3/2001Pyrimethamine and Sulfadoxine for Treatment of Autoimmune Lymphoproliferative SyndromePilot Study of Pyrimethamine and Sulfadoxine (Fansidar) for the Treatment of Individuals With the Autoimmune Lymphoproliferative Syndrome (ALPS)Autoimmune Disease;Lymphoproliferative DisorderDrug: Fansidar (pyrimethamine and sulfadoxine)National Institute of Allergy and Infectious Diseases (NIAID)NULLCompletedN/AN/ABoth8Phase 1United States

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