Campath    (DrugBank: -)

12 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3
20副腎白質ジストロフィー1
49全身性エリテマトーデス1
51全身性強皮症2
60再生不良性貧血8
62発作性夜間ヘモグロビン尿症1
65原発性免疫不全症候群4
164眼皮膚白皮症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
283後天性赤芽球癆1
285ファンコニ貧血2
326大理石骨病2

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.
3 / 784 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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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
2NCT01043640
(ClinicalTrials.gov)
December 20095/1/2010Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersAllogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic DisordersMucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal DisordersDrug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate MofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A21 YearsAll46Phase 2United States
3NCT00668564
(ClinicalTrials.gov)
March 200825/4/2008Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTreatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell TransplantationHurler's Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Sphingolipidoses;Krabbe Disease;Wolman's Disease;Niemann-Pick Disease Type B;Niemann-Pick Disease, Type CProcedure: Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: Campath-1H;Drug: BusulfanMasonic Cancer Center, University of MinnesotaNULLTerminatedN/A21 YearsAll18Phase 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_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1NCT01043640
(ClinicalTrials.gov)
December 20095/1/2010Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersAllogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic DisordersMucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal DisordersDrug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate MofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A21 YearsAll46Phase 2United 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
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT00684255
(ClinicalTrials.gov)
August 200722/5/2008Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)Systemic Lupus Erythematosus;Systemic SclerosisProcedure: Reduced Intensity Allogeneic Transplant;Drug: Fludarabine;Drug: Busulfan;Drug: CampathNew York Medical CollegeNULLTerminated7 Years50 YearsAll1Phase 1United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 466 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT01639573
(ClinicalTrials.gov)
April 201118/6/2012Campath-1h Phase I/II Pilot Trial as Immunoablative Therapy for Refractory Systemic SclerosisCampath-1h as Immunoablative Therapy for Children and Adolescents With Treatment Refractory Systemic SclerosisSclerodermaDrug: CampathChildren's Hospital Los AngelesNULLWithdrawn8 Years18 YearsAll0Phase 1;Phase 2United States
2NCT00684255
(ClinicalTrials.gov)
August 200722/5/2008Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc)Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc)Systemic Lupus Erythematosus;Systemic SclerosisProcedure: Reduced Intensity Allogeneic Transplant;Drug: Fludarabine;Drug: Busulfan;Drug: CampathNew York Medical CollegeNULLTerminated7 Years50 YearsAll1Phase 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.
8 / 218 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT00455312
(ClinicalTrials.gov)
August 200730/3/2007Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAAHematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: MethylprednisoloneMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll36Phase 2;Phase 3United States
2NCT00578266
(ClinicalTrials.gov)
February 200717/12/2007Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic AnemiaAllogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related DonorsAnemia, AplasticDrug: Cyclophosphamide,Campath IH and TBIMayo ClinicNULLCompletedN/A60 YearsAll8Phase 1United States
3NCT00195624
(ClinicalTrials.gov)
September 15, 200516/9/2005Alemtuzumab to Treat Severe Aplastic AnemiaA Pilot Study of Alemtuzumab (Campath) in Patients With Relapsed or Refractory Severe Aplastic AnemiaSevere Aplastic Anemia, Refractory;Severe Aplastic Anemia, RelapseBiological: Alemtuzumab (Campath );Drug: CyclosporineNational Heart, Lung, and Blood Institute (NHLBI)NULLCompleted2 Years110 YearsAll47Phase 2United States
4NCT00065260
(ClinicalTrials.gov)
July 200318/7/2003Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic AnemiaA Randomized Trial of Immunosuppression in Aplastic Anemia Patients With Refractory Pancytopenia or Suboptimal Hematologic Response After h-ATG/CsA TreatmentAplastic AnemiaDrug: Campath-1H;Drug: r-ATG;Drug: CsANational Heart, Lung, and Blood Institute (NHLBI)NULLCompleted2 YearsN/AAll54Phase 2United States
5NCT00533923
(ClinicalTrials.gov)
December 200220/9/2007Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic DisordersNonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic DisordersAML;ALL;CLL;Myelodysplastic Syndrome;Non-Hodgkin's Lymphoma;Hodgkin's Lymphoma;Multiple Myeloma;Aplastic Anemia;Myeloproliferative DisorderDrug: Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSFBeth Israel Deaconess Medical CenterBayerCompletedN/A65 YearsBoth25Phase 2United States
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
6NCT00578903
(ClinicalTrials.gov)
February 200219/12/2007Allogeneic Stem Cell Transplant for Patients With Severe Aplastic AnemiaAllogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD)Aplastic AnemiaDrug: Cytoxan;Drug: Campath;Radiation: Total Body Irradiation (TBI);Drug: FK-506;Drug: Methotrexate;Procedure: Stem cell infusionBaylor College of MedicineThe Methodist Hospital System;Texas Children's Hospital;Center for Cell and Gene Therapy, Baylor College of MedicineTerminatedN/A60 YearsAll22Phase 2United States
7NCT00590460
(ClinicalTrials.gov)
July 200126/12/2007Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi AnemiaCd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi AnemiaFanconi Anemia;Severe Aplastic AnemiaBiological: CAMPATH-1H;Biological: Anti-CD45;Drug: Fludarabine;Procedure: Stem cell infusionBaylor College of MedicineThe Methodist Hospital System;Center for Cell and Gene Therapy, Baylor College of MedicineTerminatedN/AN/AAll5Phase 1;Phase 2United States
8NCT00004143
(ClinicalTrials.gov)
September 199910/12/1999Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure SyndromesAllogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure SyndromesSickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell AplasiaDrug: Campath, Chemo and/or TBI Allo SCTDavid Rizzieri, MDNULLCompleted18 YearsN/AAll2Phase 2United 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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 202 trial found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT00004143
(ClinicalTrials.gov)
September 199910/12/1999Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure SyndromesAllogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure SyndromesSickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell AplasiaDrug: Campath, Chemo and/or TBI Allo SCTDavid Rizzieri, MDNULLCompleted18 YearsN/AAll2Phase 2United States

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.
4 / 413 trials found
No.TrialIDDate_
enrollment
Date_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1NCT02629120
(ClinicalTrials.gov)
December 17, 201510/12/2015High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseHigh Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous DiseaseChronic Granulomatous Disease TransplantDrug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cellsNational Institute of Allergy and Infectious Diseases (NIAID)NULLRecruiting4 Years65 YearsAll50Phase 1;Phase 2United States
2NCT00579137
(ClinicalTrials.gov)
October 200719/12/2007Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency DisordersCD45 and Alemtuzumab Monoclonal Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Severe Combined Immunodeficiency Disease (SCID) And Other Primary Immunodeficiency DisordersSevere Combined Immunodeficiency Disease;Severe Primary Immunodeficiency Disorder;Undefined T Cell Deficiency Disorder;Wiskott-Aldrick SyndromeBiological: Campath -1H;Drug: Fludarabine;Biological: Anti-CD45;Procedure: Stem cell infusionBaylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of Medicine;Texas Children's HospitalTerminatedN/AN/AAll3Phase 1;Phase 2United States
3NCT00455312
(ClinicalTrials.gov)
August 200730/3/2007Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAAHematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: MethylprednisoloneMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll36Phase 2;Phase 3United States
4NCT00176852
(ClinicalTrials.gov)
June 200212/9/2005Stem Cell Transplant for HemoglobinopathyAllogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed ChimerismSickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond SyndromeDrug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusionMasonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramCompletedN/A50 YearsAll22Phase 2;Phase 3United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 15 trial found
No.TrialIDDate_
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1NCT00176865
(ClinicalTrials.gov)
August 200212/9/2005Stem Cell Transplant for Immunologic or Histiocytic DisordersAllogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed ChimerismHemophagocytic Lymphohistiocytosis;X-Linked Lymphoproliferative Disorders;Chediak-Higashi Syndrome;Griscelli Syndrome;Immunologic Deficiency Syndromes;Langerhans-Cell HistiocytosisProcedure: Stem Cell Transplant;Drug: Fludarabine;Drug: Melphalan;Drug: Anti-thymocyte globulin (ATG);Drug: Campath 1H;Drug: Cyclosporin A;Drug: Mycophenolate mofetil;Drug: Intravenous immunoglobulin (IVIG)Masonic Cancer Center, University of MinnesotaNULLCompletedN/A35 YearsAll19Phase 2United 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_
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Public_titleScientific_titleConditionInterventionPrimary_
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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

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 17 trial found
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1NCT00004143
(ClinicalTrials.gov)
September 199910/12/1999Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure SyndromesAllogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure SyndromesSickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell AplasiaDrug: Campath, Chemo and/or TBI Allo SCTDavid Rizzieri, MDNULLCompleted18 YearsN/AAll2Phase 2United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 56 trials found
No.TrialIDDate_
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1NCT00586274
(ClinicalTrials.gov)
March 200221/12/2007Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCTA Phase I Study Evaluating The Use Of Rft5-Dga To Deplete Alloreactive Cells For Patients With Fanconi Anemia After Haploidentical Stem Cell TransplantationFANCONI ANEMIAProcedure: CD34 selected haploidentical PBSCT;Drug: Fludarabine;Biological: T cell infusion;Biological: Campath 1h;Biological: anti-CD45Baylor College of MedicineUTSW-Dallas;Center for Cell and Gene Therapy, Baylor College of MedicineTerminatedN/A64 YearsBoth1Phase 1United States
2NCT00590460
(ClinicalTrials.gov)
July 200126/12/2007Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi AnemiaCd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi AnemiaFanconi Anemia;Severe Aplastic AnemiaBiological: CAMPATH-1H;Biological: Anti-CD45;Drug: Fludarabine;Procedure: Stem cell infusionBaylor College of MedicineThe Methodist Hospital System;Center for Cell and Gene Therapy, Baylor College of MedicineTerminatedN/AN/AAll5Phase 1;Phase 2United 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.
2 / 20 trials found
No.TrialIDDate_
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1NCT00775931
(ClinicalTrials.gov)
August 200816/10/2008Allogeneic Transplantation For Severe OsteopetrosisAllogeneic Hematopoietic Stem Cell Transplantation For Severe OsteopetrosisSevere OsteopetrosisProcedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A45 YearsAll7Phase 2;Phase 3United States
2NCT00638820
(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