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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2
20副腎白質ジストロフィー1
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1
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。）1
326大理石骨病1

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
1NCT01626092
(ClinicalTrials.gov)		July 11, 201220/6/2012Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersTreatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)Lysosomal Storage Disease;Peroxisomal DisorderDrug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A55 YearsAll3N/AUnited States
2NCT00383448
(ClinicalTrials.gov)		September 200629/9/2006HSCT for High Risk Inherited Inborn ErrorsTreatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell TransplantationAdrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Tay Sachs Disease;Sandhoffs Disease;Wolman Disease;I-Cell Disease;Sanfilippo Syndrome;GM1 GangliosidosisDrug: Clofarabine;Procedure: Total body Irradiation;Drug: Melphalan;Biological: Hematopoietic Stem Cell Transplantation;Drug: Alemtuzumab;Drug: mycophenylate mofetil;Device: Cyclosporine A;Drug: HydroxyureaMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll38Phase 2United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 49 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT00383448
(ClinicalTrials.gov)		September 200629/9/2006HSCT for High Risk Inherited Inborn ErrorsTreatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell TransplantationAdrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Tay Sachs Disease;Sandhoffs Disease;Wolman Disease;I-Cell Disease;Sanfilippo Syndrome;GM1 GangliosidosisDrug: Clofarabine;Procedure: Total body Irradiation;Drug: Melphalan;Biological: Hematopoietic Stem Cell Transplantation;Drug: Alemtuzumab;Drug: mycophenylate mofetil;Device: Cyclosporine A;Drug: HydroxyureaMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll38Phase 2United States

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.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02925351
(ClinicalTrials.gov)		January 25, 20168/9/2016Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory DiseasesThe Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory DiseasesAutoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu ArteritisProcedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission TomographyJonsson Comprehensive Cancer CenterNULLCompleted18 YearsN/AAll8Early Phase 1United 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.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02925351
(ClinicalTrials.gov)		January 25, 20168/9/2016Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory DiseasesThe Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory DiseasesAutoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu ArteritisProcedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission TomographyJonsson Comprehensive Cancer CenterNULLCompleted18 YearsN/AAll8Early Phase 1United 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.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02925351
(ClinicalTrials.gov)		January 25, 20168/9/2016Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory DiseasesThe Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory DiseasesAutoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu ArteritisProcedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission TomographyJonsson Comprehensive Cancer CenterNULLCompleted18 YearsN/AAll8Early Phase 1United 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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 218 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01500161
(ClinicalTrials.gov)		November 201118/11/2011Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-MatchA Phase II Study Of Pooled Unrelated Donor Umbilical Cord Blood (UCB) Transplant For Patients With Hematologic Malignancies Needing Allogeneic Stem Cell Transplant But Do Not Have A Related HLA-Matched DonorAcute Myelogenous Leukemia;Acute Lymphocytic Leukemia;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia;Hodgkins Disease;Non-Hodgkins Lymphoma;Aplastic Anemia;Multiple Myeloma;Myelodysplastic SyndromeDrug: Busulfan;Drug: Clofarabine;Drug: Fludarabine;Drug: Melphalan;Drug: Carmustine;Drug: Etoposide;Drug: CytarabineTexas Oncology Cancer CenterNULLTerminated18 Years65 YearsBoth1Phase 2United States

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.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02925351
(ClinicalTrials.gov)		January 25, 20168/9/2016Fluorine F 18 Clofarabine PET/CT in Imaging Patients With Autoimmune or Inflammatory DiseasesThe Biodistribution of 18F-Clofarabine in Patients With Autoimmune and Inflammatory DiseasesAutoimmune Disease;Crohn Disease;Inflammatory Disorder;Rheumatoid Arthritis;Systemic Lupus Erythematosus;Takayasu ArteritisProcedure: Computed Tomography;Radiation: Fluorine F 18 Clofarabine;Procedure: Positron Emission TomographyJonsson Comprehensive Cancer CenterNULLCompleted18 YearsN/AAll8Early Phase 1United 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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 38 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT01626092
(ClinicalTrials.gov)		July 11, 201220/6/2012Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersTreatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)Lysosomal Storage Disease;Peroxisomal DisorderDrug: Campath-1H;Drug: Clofarabine;Drug: Melphalan;Radiation: Total Body Irradiation with Marrow Boosting;Biological: Hematopoietic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate mofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A55 YearsAll3N/AUnited States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 20 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT00638820
(ClinicalTrials.gov)		September 200711/3/2008Reduced Intensity AlloTransplant For OsteopetrosisReduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected GraftOsteopetrosisProcedure: Stem Cell or Umbilical Cord Blood Transplantation;Drug: Campath, Busulfan, Clofarabine;Procedure: Total Lymphoid IrradiationMasonic Cancer Center, University of MinnesotaNULLTerminatedN/A45 YearsAll3Phase 2United States

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