DDrare: Database of Drug Development for Rare Diseases

Other: eculizumab (DrugBank: Eculizumab)

8 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
11	重症筋無力症	0
13	多発性硬化症／視神経脊髄炎	0
14	慢性炎症性脱髄性多発神経炎／多巣性運動ニューロパチー	0
61	自己免疫性溶血性貧血	0
62	発作性夜間ヘモグロビン尿症	0
109	非典型溶血性尿毒症症候群	1
222	一次性ネフローゼ症候群	0
223	一次性膜性増殖性糸球体腎炎	0

11. 重症筋無力症　［臨床試験数：226，薬物数：172（DrugBank：45），標的遺伝子数：43，標的パスウェイ数：118］

Searched query = "Myasthenia", "MG"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 226 trial found

13. 多発性硬化症／視神経脊髄炎　［臨床試験数：3,050，薬物数：2,147（DrugBank：348），標的遺伝子数：244，標的パスウェイ数：228］

Searched query = "Multiple sclerosis/Neuromyelitis optica", "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 3,050 trial found

14. 慢性炎症性脱髄性多発神経炎／多巣性運動ニューロパチー　［臨床試験数：145，薬物数：139（DrugBank：23），標的遺伝子数：12，標的パスウェイ数：21］

Searched query = "Chronic inflammatory demyelinating polyneuropathy", "Chronic inflammatory demyelinating poly (radiculo) neuropathy", "Multifocal motor neuropathy", "CIDP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 145 trial found

61. 自己免疫性溶血性貧血　［臨床試験数：90，薬物数：72（DrugBank：23），標的遺伝子数：19，標的パスウェイ数：147］

Searched query = "Autoimmune hemolytic anemia", "AIHA", "WAIHA", "Cold agglutinin disease", "CAD", "Paroxysmal cold hemoglobinuria", "MAIHA", "Evans syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 90 trial found

62. 発作性夜間ヘモグロビン尿症　［臨床試験数：202，薬物数：120（DrugBank：25），標的遺伝子数：15，標的パスウェイ数：90］

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 202 trial found

109. 非典型溶血性尿毒症症候群　［臨床試験数：95，薬物数：27（DrugBank：5），標的遺伝子数：2，標的パスウェイ数：9］

Searched query = "Atypical hemolytic uremic syndrome", "aHUS"
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 / 95 trial found

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	NCT02614898 (ClinicalTrials.gov)	November 4, 2015	24/11/2015	Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment	Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment	Atypical Hemolytic Uremic Syndrome	Other: Eculizumab	Alexion Pharmaceuticals	NULL	Terminated	N/A	N/A	All	67		United States;Australia;Germany;United Kingdom

222. 一次性ネフローゼ症候群　［臨床試験数：234，薬物数：241（DrugBank：78），標的遺伝子数：59，標的パスウェイ数：185］

Searched query = "Primary nephrotic syndrome", "Minimal change nephrotic syndrome", "MCNS", "Membranous nephropathy", "Focal segmental glomerulosclerosis", "FSGS", "Membranoproliferative glomerulonephritis", "MPGN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 234 trial found

223. 一次性膜性増殖性糸球体腎炎　［臨床試験数：18，薬物数：14（DrugBank：7），標的遺伝子数：7，標的パスウェイ数：14］

Searched query = "Primary membranoproliferative glomerulonephritis", "Dense deposit disease"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 18 trial found