Hematopoietic stem cell transplantation    (DrugBank: -)

12 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2
13多発性硬化症/視神経脊髄炎12
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2
19ライソゾーム病8
20副腎白質ジストロフィー2
35天疱瘡1
46悪性関節リウマチ1
49全身性エリテマトーデス2
51全身性強皮症8
96クローン病5
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
285ファンコニ貧血13

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 226 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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PhaseCountries
1NCT00716066
(ClinicalTrials.gov)
June 200815/7/2008Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune DiseasesHigh-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic DiseasesAutoimmune Disease;Neurologic Autoimmune Disease;Autologous Transplant Autoimmune;Multiple Sclerosis Transplant;MS Stem Cell Transplant;Multiple Sclerosis Stem Cell Transplant;Stiff Person Syndrome;HCT for Neurologic Autoimmune Disorders;CIDP Transplant;Myasthenia Gravis Transplant;Autoimmune Nervous System Disorder;Central Nervous System Vasculitis;Cerebellar Degeneration;Chronic Inflammatory Demyelinating Polyneuropathy;Lambert Eaton Myasthenic Syndrome;Myasthenia Gravis;Neuromyelitis Optica;Opsoclonus Myoclonus Syndrome;Rasmussen Subacute EncephalitisBiological: Anti-Thymocyte Globulin;Procedure: Autologous Hematopoietic Stem Cell Transplantation;Drug: Carmustine;Drug: Cytarabine;Drug: Etoposide;Other: Laboratory Biomarker Analysis;Drug: Melphalan;Procedure: Peripheral Blood Stem Cell Transplantation;Drug: Prednisone;Procedure: Syngeneic Bone Marrow TransplantationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)RecruitingN/A71 YearsAll40Phase 2United States
2NCT00424489
(ClinicalTrials.gov)
February 200218/1/2007Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia GravisHematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia GravisMyasthenia GravisBiological: Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: ATG (rabbit);Drug: Mesna;Drug: Methylprednisolone;Drug: G-CSFNorthwestern UniversityNULLTerminated15 Years65 YearsAll9Phase 1United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
12 / 3,050 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
1EUCTR2016-001166-29-DE
(EUCTR)
18/03/202012/08/2019A randomised controlled trial to compare ocrelizumab or alemtuzumab with autologous hematopoietic stem cell transplantation (aHSCT) in high inflammatory multiple sclerosis (COAST)A randomised controlled trial to compare ocrelizumab or alemtuzumab with autologous hematopoietic stem cell transplantation (aHSCT) in high inflammatory multiple sclerosis (COAST) - COAST relapsing-remitting multiple sclerosis
MedDRA version: 21.1;Level: PT;Classification code 10063399;Term: Relapsing-remitting multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Lemtrada
Product Name: alemtuzumab
INN or Proposed INN: alemtuzumab
Other descriptive name: ALEMTUZUMAB
Trade Name: Ocrevus
Product Name: ocrelizumab
INN or Proposed INN: ocrelizumab
Other descriptive name: OCRELIZUMAB
Product Name: autologous hematopoietic stem cells
Product Code: aHSC
University Medical Centre Hamburg-EppendorfNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 2Germany
2NCT04047628
(ClinicalTrials.gov)
December 19, 20195/8/2019Best Available Therapy Versus Autologous Hematopoetic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)A Multicenter Randomized Controlled Trial of Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis (ITN077AI)Relapsing Multiple Sclerosis;Relapsing Remitting Multiple Sclerosis;Secondary Progressive Multiple SclerosisProcedure: Autologous Hematopoietic Stem Cell Transplantation;Biological: Best Available Therapy (BAT)National Institute of Allergy and Infectious Diseases (NIAID)Immune Tolerance Network (ITN);Blood and Marrow Transplant Clinical Trials Network;PPD;Rho Federal Systems Division, Inc.Recruiting18 Years55 YearsAll156Phase 3United States;United Kingdom
3EUCTR2017-001362-25-NL
(EUCTR)
30/09/201910/09/2019 Randomized study with stem cell transplantation versus standard treatment with alemtuzumab, cladribine or ocrelizumab in patients with relapsing remitting multiple sclerosis. Randomized autologous hematopoietic stem cell transplantation versus alemtuzumab, cladribine or ocrelizumab for patients with relapsing remitting Multiple Sclerosis - RAM-MS Relapsing remitting multiple sclerosis.
MedDRA version: 21.1;Level: PT;Classification code 10063399;Term: Relapsing-remitting multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Helse Bergen HF, Haukeland University HospitalNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
100Phase 3Denmark;Netherlands
4NCT04203017
(ClinicalTrials.gov)
June 1, 201930/10/2019Fecal Microbiota Transplantation After Autologous HSCT in Patients With Multiple SclerosisAllogeneic Fecal Microbiota Transplantation as a Consolidation Treatment After Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple SclerosisMultiple SclerosisBiological: allogeneic fecal microbiotaSt. Petersburg State Pavlov Medical UniversityNULLRecruiting18 Years55 YearsAll20Phase 1Russian Federation
5EUCTR2017-001362-25-DK
(EUCTR)
27/06/201821/03/2018 Randomized study with stem cell transplantation versus standard treatment with alemtuzumab, cladribine or ocrelizumab in patients with relapsing remitting multiple sclerosis. Randomized autologous hematopoietic stem cell transplantation versus alemtuzumab, cladribine or ocrelizumab for patients with relapsing remitting Multiple Sclerosis - RAM-MS Relapsing remitting multiple sclerosis.
MedDRA version: 20.0;Level: PT;Classification code 10063399;Term: Relapsing-remitting multiple sclerosis;System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Helse Bergen HF, Haukeland University HospitalNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
100Phase 3Denmark;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
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agemax
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size
PhaseCountries
6NCT03477500
(ClinicalTrials.gov)
March 21, 201813/2/2018RCT Comparing Autologous Hematopoietic Stem Cell Transplantation Versus Alemtuzumab in MSRandomized Autologous Hematopoietic Stem Cell Transplantation Versus Alemtuzumab for Patients With Relapsing Remitting Multiple SclerosisMultiple SclerosisDrug: Cyclophosphamide and ATG;Drug: AlemtuzumabHaukeland University HospitalNULLRecruiting18 Years50 YearsAll100Phase 3Denmark;Netherlands;Norway;Sweden
7NCT03113162
(ClinicalTrials.gov)
May 29, 201510/4/2017Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) for Multiple SclerosisEvaluation of the Safety and Efficacy of Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple SclerosisMultiple SclerosisBiological: Autologous Hematopoietic Stem Cell;Drug: BEAM RegimenMakati Medical CenterNULLRecruiting18 Years60 YearsAll15Phase 1Philippines
8NCT03133403
(ClinicalTrials.gov)
November 201321/4/2016Hematopoietic Stem Cell Therapy for Inflammatory Multiple Sclerosis Failing Alternate Approved TherapyHematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Alternate Approved Therapy: A Randomized StudyMultiple Sclerosis;Multiple Sclerosis, Relapsing-RemittingDrug: Tecfidera (BG12);Drug: Gilenya;Drug: Tysabri®;Drug: Avonex/Betaseron/Copaxone/Rebif;Procedure: Hematopoietic stem cell transplantation (HSCT)Sheffield Teaching Hospitals NHS Foundation TrustNULLRecruiting18 Years55 YearsAll5Phase 2;Phase 3United Kingdom
9NCT00787722
(ClinicalTrials.gov)
October 10, 200931/10/2008Hematopoietic Stem Cell Transplant in Devic's DiseaseTrial of High Dose Immunosuppressive Therapy With Hematopoietic Stem Cell Support in Devic's DiseaseDevic's DiseaseProcedure: Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: G-CSF;Drug: rATG;Drug: Mesna;Drug: Rituximab;Drug: MethylprednisoloneNorthwestern UniversityNULLCompleted16 Years65 YearsAll13Phase 1;Phase 2United States
10NCT00716066
(ClinicalTrials.gov)
June 200815/7/2008Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune DiseasesHigh-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic DiseasesAutoimmune Disease;Neurologic Autoimmune Disease;Autologous Transplant Autoimmune;Multiple Sclerosis Transplant;MS Stem Cell Transplant;Multiple Sclerosis Stem Cell Transplant;Stiff Person Syndrome;HCT for Neurologic Autoimmune Disorders;CIDP Transplant;Myasthenia Gravis Transplant;Autoimmune Nervous System Disorder;Central Nervous System Vasculitis;Cerebellar Degeneration;Chronic Inflammatory Demyelinating Polyneuropathy;Lambert Eaton Myasthenic Syndrome;Myasthenia Gravis;Neuromyelitis Optica;Opsoclonus Myoclonus Syndrome;Rasmussen Subacute EncephalitisBiological: Anti-Thymocyte Globulin;Procedure: Autologous Hematopoietic Stem Cell Transplantation;Drug: Carmustine;Drug: Cytarabine;Drug: Etoposide;Other: Laboratory Biomarker Analysis;Drug: Melphalan;Procedure: Peripheral Blood Stem Cell Transplantation;Drug: Prednisone;Procedure: Syngeneic Bone Marrow TransplantationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)RecruitingN/A71 YearsAll40Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
11EUCTR2006-002562-19-IT
(EUCTR)
21/04/200624/01/2007Immunoablation with Cyclophosphamide at high dosage and Rabbit antithymoglobulin followed by autologous hematopoietic stem cell transplantation in severe multiple sclerosis - LIGHTImmunoablation with Cyclophosphamide at high dosage and Rabbit antithymoglobulin followed by autologous hematopoietic stem cell transplantation in severe multiple sclerosis - LIGHT VERIFICARE Patients with multiple sclerosis, age between 18 and 50
MedDRA version: 6.1;Level: PT;Classification code 10028245
Trade Name: L04AA04 - IMMUNOGLOBULINA ANTITIMOCITARIA (DI CONIGLIO) IMMUNOGLOBULINA DI CONIGLIO ANTITIMOCITI UMANI THYMOGLOBULINE
Product Name: Thymoglobuline
INN or Proposed INN: Antithymocyte
Trade Name: Endoxan - ciclofosfamide
Product Name: Endoxan
INN or Proposed INN: Cyclophosfamide
Dipartimento di Neuroscienze Oftalmologia e Genetica DINOG - Universita di GenovaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Italy
12NCT00278655
(ClinicalTrials.gov)
June 200316/1/2006Hematopoietic Stem Cell Therapy for Patients With Multiple SclerosisHematopoietic Stem Cell Therapy for Patients With Inflammatory Multiple Sclerosis Failing Interferon Therapy: A Phase II Multi-Center TrialMultiple SclerosisBiological: Hematopoietic stem cell transplantationNorthwestern UniversityNULLTerminated18 Years50 YearsAll21Phase 1;Phase 2United States

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: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 145 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00716066
(ClinicalTrials.gov)
June 200815/7/2008Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune DiseasesHigh-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic DiseasesAutoimmune Disease;Neurologic Autoimmune Disease;Autologous Transplant Autoimmune;Multiple Sclerosis Transplant;MS Stem Cell Transplant;Multiple Sclerosis Stem Cell Transplant;Stiff Person Syndrome;HCT for Neurologic Autoimmune Disorders;CIDP Transplant;Myasthenia Gravis Transplant;Autoimmune Nervous System Disorder;Central Nervous System Vasculitis;Cerebellar Degeneration;Chronic Inflammatory Demyelinating Polyneuropathy;Lambert Eaton Myasthenic Syndrome;Myasthenia Gravis;Neuromyelitis Optica;Opsoclonus Myoclonus Syndrome;Rasmussen Subacute EncephalitisBiological: Anti-Thymocyte Globulin;Procedure: Autologous Hematopoietic Stem Cell Transplantation;Drug: Carmustine;Drug: Cytarabine;Drug: Etoposide;Other: Laboratory Biomarker Analysis;Drug: Melphalan;Procedure: Peripheral Blood Stem Cell Transplantation;Drug: Prednisone;Procedure: Syngeneic Bone Marrow TransplantationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)RecruitingN/A71 YearsAll40Phase 2United States
2NCT00278629
(ClinicalTrials.gov)
February 21, 200516/1/2006Hematopoietic Stem Cell Transplantation in Chronic Inflammatory Demyelinating PolyneuropathyNon-myeloablative Autologous Hematopoietic Stem Cell Transplantation in Patients With Chronic Inflammatory Demyelinating Polyneuropathy: A Phase II TrialChronic Inflammatory Demyelinating PolyneuropathyBiological: hematopoietic stem cell transplantationNorthwestern UniversityNULLCompleted18 Years65 YearsAll80Phase 2United States

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.
8 / 784 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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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
2NCT01572636
(ClinicalTrials.gov)
March 28, 20124/4/2012Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler SyndromeMT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).Mucopolysaccharidosis Type IH;MPS I;Hurler SyndromeDrug: LaronidaseMasonic Cancer Center, University of MinnesotaNULLTerminatedN/AN/AAll20United States
3NCT01043640
(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
4NCT01303146
(ClinicalTrials.gov)
October 200823/2/2011Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantationand Safety of METAZYM (Recombinant Human Arylsulfatase A or rhASA) for the Treatment of Patients With Late Infantile MLD Who Had Previously Hematopoietic Stem Cell TransplantationMetachromatic LeukodystrophyDrug: rhARSAAssistance Publique - Hôpitaux de ParisEuropean Leukodystrophy Association;Zymenex A/S;ShireCompleted6 MonthsN/ABoth1Phase 2France
5NCT00668564
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
6NCT00383448
(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
7NCT00176891
(ClinicalTrials.gov)
March 200412/9/2005Stem Cell Transplant w/Laronidase for HurlerPhase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)Mucopolysaccharidosis I;Hurler SyndromeProcedure: Stem Cell Transplant;Drug: Laronidase ERTMasonic Cancer Center, University of MinnesotaNULLCompletedN/A7 YearsAll25Phase 2United States
8NCT00176917
(ClinicalTrials.gov)
May 199912/9/2005Stem Cell Transplantation for HurlerHematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Mannosidosis;Mucolipidosis Type II (I-cell Disease)Procedure: Stem Cell Transplant;Drug: Busulfan, Cyclophosphamide, ATGMasonic Cancer Center, University of MinnesotaNULLCompletedN/AN/AAll41Phase 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.
2 / 49 trials found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Inclusion_
agemin
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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
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

35. 天疱瘡 [臨床試験数:87,薬物数:114(DrugBank:37),標的遺伝子数:17,標的パスウェイ数:158
Searched query = "Pemphigus"
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 / 87 trial found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT00278642
(ClinicalTrials.gov)
September 200216/1/2006Hematopoietic Stem Cell Support in Patients With Autoimmune Bullous Skin DisordersHigh Dose Cyclophosphamide & ATG With Hematopoietic Stem Cell Support in Patients With Autoimmune Bullous Skin Disorders: A Phase I TrialPemphigusBiological: Hematopoietic stem cell transplantationRichard Burt, MDNULLTerminated18 Years60 YearsBoth1Phase 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
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sponsor
Recruitment_
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1NCT00282412
(ClinicalTrials.gov)
September 200224/1/2006Rheumatoid Arthritis:Tolerance Induction by Mixed ChimerismRheumatoid Arthritis: Tolerance Induction by Mixed ChimerismRheumatoid ArthritisBiological: Hematopoietic Stem Cell TransplantationNorthwestern UniversityNULLTerminated18 Years60 YearsBoth4Phase 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.
2 / 827 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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agemax
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PhaseCountries
1NCT00278538
(ClinicalTrials.gov)
September 23, 200515/1/2006Cyclophosphamide and Rabbit Antithymocyte Globulin (rATG)/Rituximab in Patients With Systemic Lupus ErythematosusCyclophosphamide and rATG/Rituximab in Patients With Systemic Lupus Erythematosus: Phase II TrialSystemic Lupus ErythematosusBiological: Hematopoietic stem cell transplantationNorthwestern UniversityNULLCompleted15 Years60 YearsAll32Phase 2United States
2NCT00076752
(ClinicalTrials.gov)
January 30, 20042/2/2004Lymphocyte Depletion and Stem Cell Transplantation to Treat Severe Systemic Lupus ErythematosusA Pilot Study of Intensified Lymphodepletion Followed by Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Systemic Lupus ErythematosusLupus Erythematosus, SystemicDrug: fludarabine phosphate;Drug: cyclophosphamide;Biological: Rituxan (rituximab);Biological: filgrastim;Drug: methylprednisolone;Other: immunologic technique;Other: laboratory biomarker analysis;Procedure: autologous hematopoietic stem cell transplantation;Drug: Diphenhydramine;Drug: MesnaNational Cancer Institute (NCI)NULLCompleted15 Years40 YearsAll9Phase 2United 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.
8 / 466 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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agemin
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agemax
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PhaseCountries
1EUCTR2019-004718-32-NL
(EUCTR)
09/10/202009/10/2020Stem cell transplantation in diffuse cutaneous systemic sclerosis: upfront therapy or rescue therapy after failure of immunosuppressants?Upfront autologous hematopoietic stem cell transplantation versus immunosuppressive medication in early diffuse cutaneous systemic sclerosis: an international multicentre, open-label, randomized controlled trial Diffuse cutaneous systemic sclerosis according to the ACR/EULAR criteria;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Trade Name: Mycophenolate mofetilUMC UtrechtNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 4Netherlands
2NCT04380831
(ClinicalTrials.gov)
September 22, 202030/4/2020TBI Using IMRT and Cyclophosphamide Prior to Stem Cell Transplant for the Treatment of Severe Systemic SclerosisPilot Study of Total Body Irradiation Using Intensity Modulated Radiation Therapy (IMRT) and Cyclophosphamide Conditioning Regimen Prior to Autologous Hematopoietic Cell Transplantation in Patients With Severe Systemic SclerosisSystemic SclerodermaProcedure: Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Radiation: Intensity-Modulated Radiation Therapy;Procedure: Total-Body IrradiationCity of Hope Medical CenterNational Cancer Institute (NCI)Recruiting18 Years65 YearsAll15Early Phase 1United States
3NCT01413100
(ClinicalTrials.gov)
September 15, 20118/8/2011Scleroderma Treatment With Autologous Transplant (STAT) StudyA Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic SclerosisSystemic SclerodermaBiological: Anti-Thymocyte Globulin;Procedure: Autologous Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Biological: Filgrastim;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Peripheral Blood Stem Cell Transplantation;Drug: Plerixafor;Other: Quality-of-Life Assessment;Other: Questionnaire AdministrationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)Active, not recruitingN/A70 YearsAll20Phase 2United States;Canada
4JPRN-UMIN000031940
2010/09/0728/03/2018Phase 2 trial of autologous hematopoietic stem cell transplantation for severe systemic sclerosis Systemic sclerosisCyclophosphamide 2g/m2, 2 days
Harvest of autologous hematopoietic stem cells
Cyclophosphamide 50mg/kg, 4 days
Transplantation of autologous hematopoietic stem cells
Kyushu UniversityNULLComplete: follow-up complete16years-old65years-oldMale and Female24Phase 2Japan
5NCT01047072
(ClinicalTrials.gov)
January 201011/1/2010Low-Dose Conditioning Followed by Donor Stem Cell Transplant in Treating Patients With Severe Systemic SclerosisPhase II Clinical Trial of Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic SclerosisSystemic SclerodermaDrug: fludarabine phosphate;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Biological: rituximab;Drug: cyclophosphamide;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationFred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumNational Cancer Institute (NCI)Withdrawn18 Years65 YearsBoth0Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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PhaseCountries
6EUCTR2006-004598-83-GB
(EUCTR)
22/10/200824/10/2008High dose immunoablation and autologous hematopoietic stem cell transplantation versus monthly intravenous pulse therapy cyclophosphamide in severe systemic sclerosis ( ‘ASTIS’-TRIAL: Autologous Stem Cell Transplantation International Scleroderma Trial) - ASTISHigh dose immunoablation and autologous hematopoietic stem cell transplantation versus monthly intravenous pulse therapy cyclophosphamide in severe systemic sclerosis ( ‘ASTIS’-TRIAL: Autologous Stem Cell Transplantation International Scleroderma Trial) - ASTIS Severe systemic sclerosis
MedDRA version: 9.1;Level: LLT;Classification code 10042953;Term: Systemic sclerosis
Trade Name: Cyclophosphamide
Product Name: Cyclophosphamide
INN or Proposed INN: CYCLOPHOSPHAMIDE
Trade Name: Neupogen
Product Name: Filgrastim
INN or Proposed INN: FILGRASTIM
Trade Name: Thymoglobuline
Product Name: Thymoglobuline
Product Code: rbATG
INN or Proposed INN: Anti-Thymocyte Globulin, Rabbit
Other descriptive name: ANTI-HUMAN T-LYMPHOCYTE IMMUNOGLOBULIN FROM RABBITS
Trade Name: Solu-Medrone
Product Name: Methylprednisolone
INN or Proposed INN: METHYLPREDNISOLONE
Trade Name: Cyclophosphamide
Product Name: Cyclophosphamide
INN or Proposed INN: CYCLOPHOSPHAMIDE
Trade Name: Mesna
Product Name: Mesna
INN or Proposed INN: MESNA
Trade Name: Mesna
Product Name: Mesna
INN or Proposed INN: MESNA
EBMT (European group for Blood and Marrow Transplantation)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
150United Kingdom
7NCT00622895
(ClinicalTrials.gov)
September 1, 200622/2/2008Allogeneic Hematopoietic Cell Transplantation for Severe Systemic SclerosisAllogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic SclerosisSystemic Scleroderma;Severe Systemic SclerosisDrug: fludarabine phosphate;Drug: Mycophenolic Acid;Drug: tacrolimus;Radiation: total-body irradiation;Procedure: bone marrow transplantation;Procedure: reduced intensity allogeneic hematopoietic stem cell transplantation;Procedure: quality-of-life assessment;Other: laboratory biomarker analysis;Other: flow cytometry;Procedure: biopsyFred Hutchinson Cancer Research CenterNational Institute of Allergy and Infectious Diseases (NIAID)CompletedN/A70 YearsAll3Phase 1;Phase 2United States
8NCT00282425
(ClinicalTrials.gov)
May 200524/1/2006Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic SclerosisNon-myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic SclerosisSclerodermaBiological: Hematopoietic stem cell transplantationRichard Burt, MDNULLTerminated18 Years55 YearsBoth8Phase 1United 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.
5 / 2,209 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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agemax
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PhaseCountries
1NCT04224558
(ClinicalTrials.gov)
January 1, 20204/4/2018Stem Cell Transplantation in Crohn's DiseaseAutologous Hematopoietic Stem Cell Transplantation for Refractory Crohn's DiseaseCrohn DiseaseDrug: Mesna;Drug: Cyclophosphamide;Drug: Filgrastim;Procedure: Apheresis catheter placement;Procedure: Leukapheresis;Drug: Fludarabine;Drug: Methylprednisolone;Drug: Diphenhydramine;Drug: Acetaminophen;Drug: anti-thymocyte globulin (rabbit);Drug: lymphocyte immune globulin;Biological: Peripheral Blood Stem Cell Infusion;Drug: CytoxanCedars-Sinai Medical CenterNULLNot yet recruiting16 Years24 YearsAll15Phase 1;Phase 2United States
2NCT01932658
(ClinicalTrials.gov)
February 201428/6/2013Autologous Hematopoietic Stem Cell Transplantation for Crohn's Disease TreatmentAutologous Hematopoietic Stem Cell Transplantation for Crohn's Disease TreatmentCrohn's DiseaseBiological: Hematopoietic stem cell transplantationUniversity of California, Los AngelesNULLWithdrawn7 Years70 YearsBoth0Phase 0United States
3NCT02676622
(ClinicalTrials.gov)
April 201321/4/2013Autologous Stem Cell Transplant for Refractory Crohn's DiseaseA Pilot Study of Autologous Hematopoietic Stem Cell Transplantation With Post-Transplant Ultra Low-Dose IL-2 for Refractory Crohn's DiseaseCrohn's DiseaseDrug: mobilization of stem cells to prepare for Leukapheresis;Other: Leukapheresis- Collection of stem cells;Drug: Preparative regimen 4-6 weeks after Leukapheresis;Other: Stem Cell Transplant;Drug: Low-dose IL-2 administrationNationwide Children's HospitalNULLWithdrawn12 Years29 YearsAll0Phase 2NULL
4NCT01288053
(ClinicalTrials.gov)
January 201027/1/2011Non-myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Refractory Crohn's DiseaseNon-myeloablative Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Refractory Crohn's DiseaseCrohn's DiseaseBiological: Allogeneic Stem Cell TherapyNorthwestern UniversityNULLTerminated18 Years45 YearsAll9Phase 1;Phase 2United States
5NCT00271947
(ClinicalTrials.gov)
April 20052/1/2006Crohn's Disease Stem Cell TransplantationCrohn's Disease Non-myeloablative Autologous Hematopoietic Stem Cell Transplantation (CDNST) Versus Standard TherapyCrohn's DiseaseBiological: Autologous Stem Cell TransplantationNorthwestern UniversityNULLTerminated18 Years55 YearsAll1Phase 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_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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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

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.
13 / 56 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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PhaseCountries
1NCT03600909
(ClinicalTrials.gov)
May 15, 201817/7/2018A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi AnemiaA Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineFanconi Anemia;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Anti-Thymocyte Globulin (Rabbit);Device: The CliniMACS device;Drug: G-CSFMemorial Sloan Kettering Cancer CenterPediatric Brain Tumor ConsortiumRecruiting1 MonthN/AAll70Phase 2United States
2NCT01319851
(ClinicalTrials.gov)
September 201015/9/2010Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaTerminatedN/A21 YearsAll3N/AUnited States
3NCT01082133
(ClinicalTrials.gov)
October 20095/3/2010Multicenter Transplant Study for Fanconi AnemiaA Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineFanconi AnemiaDrug: Chemotherapy;Biological: Miltenyi CliniMACSChildren's Hospital Medical Center, CincinnatiNULLCompletedN/AN/ABoth45Phase 2United States
4NCT00987480
(ClinicalTrials.gov)
September 25, 200930/9/2009Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineA Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineAplastic Anemia;Leukemia;Myelodysplastic SyndromeDrug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.;Device: CliniMACS deviceMemorial Sloan Kettering Cancer CenterBoston Children's Hospital;Children's Hospital Medical Center, Cincinnati;Children's Hospital and Health System Foundation, Wisconsin;Rockefeller University;Fred Hutchinson Cancer Research CenterCompletedN/AN/AAll45Phase 2United States
5NCT01071239
(ClinicalTrials.gov)
April 200920/5/2009Hematopoietic Stem Cell Transplant for Fanconi AnemiaA Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineFanconi AnemiaDevice: CliniMACs device;Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: ATGMedical College of WisconsinMemorial Sloan Kettering Cancer CenterCompletedN/AN/AAll1Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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PhaseCountries
6NCT00856388
(ClinicalTrials.gov)
January 14, 20094/3/2009Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure DisordersA Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Fanconi Anemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncontiguous Stage II Adult Burkitt Lymphoma;Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Noncontiguous Stage II Adult Lymphoblastic Lymphoma;Noncontiguous Stage II Grade 1 Follicular Lymphoma;Noncontiguous Stage II Grade 2 Follicular Lymphoma;Noncontiguous Stage II Grade 3 Follicular Lymphoma;Noncontiguous Stage II Mantle Cell Lymphoma;Noncontiguous Stage II Marginal Zone Lymphoma;Noncontiguous Stage II Small Lymphocytic Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Relapsing Chronic Myelogenous Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Grade 1 Follicular Lymphoma;Stage III Grade 2 Follicular Lymphoma;Stage III Grade 3 Follicular Lymphoma;Stage III Mantle Cell Lymphoma;Stage III Marginal Zone Lymphoma;Stage III Small Lymphocytic Lymphoma;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Grade 1 Follicular Lymphoma;Stage IV Grade 2 Follicular Lymphoma;Stage IV Grade 3 Follicular Lymphoma;Stage IV Mantle Cell Lymphoma;Stage IV Marginal Zone Lymphoma;Stage IV Small Lymphocytic Lymphoma;Waldenström MacroglobulinemiaDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulinRoswell Park Cancer InstituteNational Cancer Institute (NCI)Completed3 Years75 YearsAll62N/AUnited States
7NCT00453388
(ClinicalTrials.gov)
February 200727/3/2007Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi AnemiaNonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding StudyAcute Myeloid Leukemia in Remission;de Novo Myelodysplastic Syndrome;Fanconi Anemia;Previously Treated Myelodysplastic SyndromeProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)CompletedN/AN/AAll6Phase 2United States;Brazil
8NCT00167206
(ClinicalTrials.gov)
March 20049/9/2005Stem Cell Transplantation for Fanconi AnemiaA Study of Thymic Shielding in Recipients of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Stem Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaProcedure: Hematopoietic Stem Cell Transplant;Procedure: Thymic Shielding During Radiation;Procedure: Total Body Irradiation;Drug: Cyclophosphamide, FludarabineMasonic Cancer Center, University of MinnesotaNULLTerminatedN/A18 YearsAll16Phase 1;Phase 2United States
9NCT00258427
(ClinicalTrials.gov)
March 26, 200222/11/2005Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi AnemiaHematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02Fanconi AnemiaBiological: anti-thymocyte globulin;Biological: filgrastim;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Biological: Hematopoietic stem cell transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A44 YearsAll14Phase 2United States
10NCT00595127
(ClinicalTrials.gov)
June 20017/1/2008Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and FludarabineA Pilot Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and FludarabineFanconi AnemiaDrug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total body irradiation (TBI)Memorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI);Miltenyi Biotec, Inc.CompletedN/AN/AAll21N/AUnited States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
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gender
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size
PhaseCountries
11NCT00630253
(ClinicalTrials.gov)
February 17, 20005/3/2008Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi AnemiaA Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaBiological: Anti-Thymocyte Globulin;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Hematopoietic Stem Cell Transplantation;Drug: Methylprednisolone;Drug: Filgrastim;Drug: Cyclosporine;Drug: Mycophenolate MofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A59 YearsAll31Phase 1;Phase 2United States
12NCT00093743
(ClinicalTrials.gov)
January 20006/10/2004Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi AnemiaLow-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter TrialAdult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Childhood Acute Myeloid Leukemia in Remission;Childhood Myelodysplastic Syndromes;Fanconi Anemia;Previously Treated Myelodysplastic SyndromesDrug: fludarabine phosphate;Drug: cyclosporine;Radiation: total-body irradiation;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Drug: mycophenolate mofetilFred Hutchinson Cancer Research CenterNational Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI)CompletedN/AN/AAll2Phase 1United States
13NCT00317876
(ClinicalTrials.gov)
June 199824/4/2006Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's AnemiaDose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi AnemiaFanconi AnemiaDrug: cyclophosphamide;Drug: cyclosporine;Drug: methotrexate;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)CompletedN/AN/ABoth25Phase 1United States;Brazil