DDrare: Database of Drug Development for Rare Diseases

Radiation: fluorine f 18 clofarabine (DrugBank: Clofarabine)

9 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	0
20	副腎白質ジストロフィー	0
40	高安動脈炎	1
46	悪性関節リウマチ	1
49	全身性エリテマトーデス	1
60	再生不良性貧血	0
96	クローン病	1
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
326	大理石骨病	0

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

40. 高安動脈炎　［臨床試験数：22，薬物数：46（DrugBank：20），標的遺伝子数：22，標的パスウェイ数：111］

Searched query = "Takayasu arteritis", "Aortitis syndrome", "Pulseless disease"
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 / 22 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	NCT02925351 (ClinicalTrials.gov)	January 25, 2016	8/9/2016	Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory Diseases	The Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory Diseases	Autoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu Arteritis	Procedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission Tomography	Jonsson Comprehensive Cancer Center	NULL	Completed	18 Years	N/A	All	8	Early Phase 1	United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 4,183 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	NCT02925351 (ClinicalTrials.gov)	January 25, 2016	8/9/2016	Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory Diseases	The Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory Diseases	Autoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu Arteritis	Procedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission Tomography	Jonsson Comprehensive Cancer Center	NULL	Completed	18 Years	N/A	All	8	Early Phase 1	United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 827 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	NCT02925351 (ClinicalTrials.gov)	January 25, 2016	8/9/2016	Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory Diseases	The Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory Diseases	Autoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu Arteritis	Procedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission Tomography	Jonsson Comprehensive Cancer Center	NULL	Completed	18 Years	N/A	All	8	Early Phase 1	United States

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

0 / 218 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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

1 / 2,209 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	NCT02925351 (ClinicalTrials.gov)	January 25, 2016	8/9/2016	Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory Diseases	The Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory Diseases	Autoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu Arteritis	Procedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission Tomography	Jonsson Comprehensive Cancer Center	NULL	Completed	18 Years	N/A	All	8	Early Phase 1	United States

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

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