DDrare: Database of Drug Development for Rare Diseases

Campath-1h (alemtuzumab) (DrugBank: Alemtuzumab)

21 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
13	多発性硬化症／視神経脊髄炎	0
15	封入体筋炎	0
19	ライソゾーム病	0
20	副腎白質ジストロフィー	0
43	顕微鏡的多発血管炎	0
44	多発血管炎性肉芽腫症	0
46	悪性関節リウマチ	0
49	全身性エリテマトーデス	0
51	全身性強皮症	0
60	再生不良性貧血	1
62	発作性夜間ヘモグロビン尿症	0
63	特発性血小板減少性紫斑病	0
65	原発性免疫不全症候群	0
85	特発性間質性肺炎	0
96	クローン病	0
164	眼皮膚白皮症	0
234	ペルオキシソーム病（副腎白質ジストロフィーを除く。）	0
283	後天性赤芽球癆	0
284	ダイアモンド・ブラックファン貧血	0
285	ファンコニ貧血	0
326	大理石骨病	0

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

15. 封入体筋炎　［臨床試験数：39，薬物数：32（DrugBank：11），標的遺伝子数：11，標的パスウェイ数：119］

Searched query = "Inclusion body myositis"
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 / 39 trial found

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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 784 trial found

20. 副腎白質ジストロフィー　［臨床試験数：49，薬物数：86（DrugBank：29），標的遺伝子数：18，標的パスウェイ数：112］

Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
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 / 49 trial found

43. 顕微鏡的多発血管炎　［臨床試験数：85，薬物数：80（DrugBank：19），標的遺伝子数：13，標的パスウェイ数：85］

Searched query = "Microscopic polyangiitis", "MPA"
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 / 85 trial found

44. 多発血管炎性肉芽腫症　［臨床試験数：84，薬物数：88（DrugBank：22），標的遺伝子数：13，標的パスウェイ数：37］

Searched query = "Wegener granulomatosis", "Multiple vasculitis granulomatous disease", "Granulomatosis with polyangiitis"
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 / 84 trial found

46. 悪性関節リウマチ　［臨床試験数：4,183，薬物数：2,538（DrugBank：401），標的遺伝子数：183，標的パスウェイ数：219］

Searched query = "Malignant rheumatoid arthritis", "Rheumatoid arthritis", "Rheumatoid arthritis with vasculitis"
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 / 4,183 trial found

49. 全身性エリテマトーデス　［臨床試験数：827，薬物数：638（DrugBank：168），標的遺伝子数：108，標的パスウェイ数：191］

Searched query = "Systemic lupus erythematosus", "SLE"
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 / 827 trial found

51. 全身性強皮症　［臨床試験数：466，薬物数：536（DrugBank：142），標的遺伝子数：110，標的パスウェイ数：210］

Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
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 / 466 trial found

60. 再生不良性貧血　［臨床試験数：218，薬物数：362（DrugBank：81），標的遺伝子数：39，標的パスウェイ数：155］

Searched query = "Aplastic anemia"
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 / 218 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	NCT00533923 (ClinicalTrials.gov)	December 2002	20/9/2007	Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders	Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders	AML;ALL;CLL;Myelodysplastic Syndrome;Non-Hodgkin's Lymphoma;Hodgkin's Lymphoma;Multiple Myeloma;Aplastic Anemia;Myeloproliferative Disorder	Drug: Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSF	Beth Israel Deaconess Medical Center	Bayer	Completed	N/A	65 Years	Both	25	Phase 2	United States

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

63. 特発性血小板減少性紫斑病　［臨床試験数：311，薬物数：185（DrugBank：39），標的遺伝子数：43，標的パスウェイ数：132］

Searched query = "Idiopathic thrombocytopenic purpura", "Primary immune thrombocytopenia"
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 / 311 trial found

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: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 413 trial found

85. 特発性間質性肺炎　［臨床試験数：514，薬物数：377（DrugBank：108），標的遺伝子数：97，標的パスウェイ数：204］

Searched query = "Idiopathic interstitial pneumonia", "IIPs", "Idiopathic pulmonary fibrosis", "IPF", "Usual interstitial pneumonia", "UIP", "Non-specific interstitial pneumonia", "NSIP", "Acute interstitial pneumonia", "AIP", "Diffuse alveolar damage", "DAD", "COP", "Organizing pneumonia", "OP", "Desquamative interstitial pneumonia", "DIP", "Respiratory bronchiolitis - associated interstitial lung disease", "RB-ILD", "Lymphocytic interstitial pneumonia", "LIP"
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 / 514 trial found

96. クローン病　［臨床試験数：2,209，薬物数：1,276（DrugBank：240），標的遺伝子数：166，標的パスウェイ数：210］

Searched query = "Crohn disease", "Terminal ileitis"
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 / 2,209 trial found

164. 眼皮膚白皮症　［臨床試験数：15，薬物数：57（DrugBank：34），標的遺伝子数：34，標的パスウェイ数：136］

Searched query = "Oculocutaneous albinism", "Hermansky-Pudlak syndrome", "Chediak-Higashi syndrome", "Griscelli 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 / 15 trial found

234. ペルオキシソーム病（副腎白質ジストロフィーを除く。）　［臨床試験数：38，薬物数：37（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：42］

Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disorder", "Zellweger syndrome", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
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 / 38 trial found

283. 後天性赤芽球癆　［臨床試験数：17，薬物数：36（DrugBank：19），標的遺伝子数：16，標的パスウェイ数：91］

Searched query = "Acquired pure red cell aplasia", "Pure red cell aplasia"
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 / 17 trial found

284. ダイアモンド・ブラックファン貧血　［臨床試験数：37，薬物数：110（DrugBank：34），標的遺伝子数：22，標的パスウェイ数：121］

Searched query = "Diamond-Blackfan anemia"
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 / 37 trial found

285. ファンコニ貧血　［臨床試験数：56，薬物数：111（DrugBank：31），標的遺伝子数：30，標的パスウェイ数：151］

Searched query = "Fanconi anemia"
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 / 56 trial found

326. 大理石骨病　［臨床試験数：20，薬物数：52（DrugBank：14），標的遺伝子数：16，標的パスウェイ数：78］

Searched query = "Osteopetrosis"
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 / 20 trial found