Fludarabine ( DrugBank: Fludarabine )


19 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1
13多発性硬化症/視神経脊髄炎4
28全身性アミロイドーシス3
36表皮水疱症5
46悪性関節リウマチ1
49全身性エリテマトーデス4
51全身性強皮症6
60再生不良性貧血51
61自己免疫性溶血性貧血1
62発作性夜間ヘモグロビン尿症6
65原発性免疫不全症候群30
85特発性間質性肺炎1
96クローン病4
164眼皮膚白皮症3
283後天性赤芽球癆2
284ダイアモンド・ブラックファン貧血7
285ファンコニ貧血22
286遺伝性鉄芽球性貧血1
326大理石骨病4

11. 重症筋無力症


臨床試験数 : 332 薬物数 : 234 - (DrugBank : 81) / 標的遺伝子数 : 45 - 標的パスウェイ数 : 127
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
1NCT04561557
(ClinicalTrials.gov)
September 22, 202010/9/2020Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Idiopathic Inflammatory Diseases of the Nervous SystemAn Open Label Clinical Trial to Evaluate the Safety and Efficacy of CT103A Cells for the Treatment of Relapsed/Refractory Antibody-associated Idiopathic Inflammatory Diseases of the Nervous SystemAutoimmune Diseases;Autoimmune Diseases of the Nervous System;Neuromyelitis Optica Spectrum Disorder;Myasthenia Gravis;Chronic Inflammatory Demyelinating Polyradiculoneuropathy;Immune-Mediated Necrotizing MyopathyBiological: CT103A cells;Drug: Cyclophosphamide and fludarabineTongji HospitalNanjing IASO Biotherapeutics Co.,LtdRecruiting18 Years75 YearsAll18Early Phase 1China

13. 多発性硬化症/視神経脊髄炎


臨床試験数 : 3,340 薬物数 : 2,163 - (DrugBank : 383) / 標的遺伝子数 : 241 - 標的パスウェイ数 : 238
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04561557
(ClinicalTrials.gov)
September 22, 202010/9/2020Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Idiopathic Inflammatory Diseases of the Nervous SystemAn Open Label Clinical Trial to Evaluate the Safety and Efficacy of CT103A Cells for the Treatment of Relapsed/Refractory Antibody-associated Idiopathic Inflammatory Diseases of the Nervous SystemAutoimmune Diseases;Autoimmune Diseases of the Nervous System;Neuromyelitis Optica Spectrum Disorder;Myasthenia Gravis;Chronic Inflammatory Demyelinating Polyradiculoneuropathy;Immune-Mediated Necrotizing MyopathyBiological: CT103A cells;Drug: Cyclophosphamide and fludarabineTongji HospitalNanjing IASO Biotherapeutics Co.,LtdRecruiting18 Years75 YearsAll18Early Phase 1China
2NCT02529839
(ClinicalTrials.gov)
October 201510/6/2015Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation (BMT) in Patients With Multiple Sclerosis (MS)Safety and Efficacy of an Immunoablative Nonmyeloablative Conditioning Protocol for Autologous Bone Marrow Transplantation in Patients With Multiple SclerosisMultiple SclerosisProcedure: Autologous bone marrow transplantation;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: AlemtuzumabHadassah Medical OrganizationNULLNot yet recruiting18 Years65 YearsBoth20N/ANULL
3NCT01679041
(ClinicalTrials.gov)
November 201229/8/2012High Dose Chemo With Stem Cell Transplant as Treatment for Multiple Sclerosis That Failed Prior TreatmentA Phase II Study of High Dose Chemotherapy With Autologous Hematopoietic Progenitor Cell Transplant for Multiple Sclerosis That Failed at Least Two Lines of TherapyMultiple SclerosisDrug: Alemtuzumab;Drug: Fludarabine;Drug: CyclophosphamideSeah Lim M.D.NULLTerminated18 Years60 YearsBoth1Phase 2United States
4EUCTR2008-005773-35-IT
(EUCTR)
15/01/2013AUTOLOUGUS STEM CELLS TRANSPLANTATION AFTER LINFOCYTE DEPLETION AS A NEW THERAPY FOR THE AGGRESSIVE AND DRUG RESISTANT FORMS OF MULTIPLE SLEROSIS IN THE ADULTS AND CHILDREN. - AUTOLOUGUS STEM CELLS TRANSPLANTATION IN SMAUTOLOUGUS STEM CELLS TRANSPLANTATION AFTER LINFOCYTE DEPLETION AS A NEW THERAPY FOR THE AGGRESSIVE AND DRUG RESISTANT FORMS OF MULTIPLE SLEROSIS IN THE ADULTS AND CHILDREN. - AUTOLOUGUS STEM CELLS TRANSPLANTATION IN SM MULTIPLE SCLEROSIS
MedDRA version: 15.1;Level: LLT;Classification code 10028053;Term: MS;System Organ Class: 100000004852;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: ENDOXAN BAXTER
INN or Proposed INN: Cyclophosphamide
Trade Name: FLUDARA
INN or Proposed INN: Fludarabine
Trade Name: THYMOGLOBULINE
INN or Proposed INN: Antithymocyte immunoglobulin (rabbit)
Trade Name: MABTHERA
INN or Proposed INN: Rituximab
AZIENDA OSPEDALIERA DI PADOVANULLNAFemale: yes
Male: yes
Phase 1Italy

28. 全身性アミロイドーシス


臨床試験数 : 267 薬物数 : 241 - (DrugBank : 77) / 標的遺伝子数 : 68 - 標的パスウェイ数 : 180
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00014235
(ClinicalTrials.gov)
December 200010/4/2001Fludarabine Phosphate and Total-Body Radiation Followed by Donor Peripheral Blood Stem Cell Transplant and Immunosuppression in Treating Patients With Hematologic MalignanciesNonmyeloablative PBSC Allografting From HLA Matched Related Donors Using Fludarabine and/or Low Dose TBI With Disease-Risk Based ImmunosuppressionAcute Myeloid Leukemia/Transient Myeloproliferative Disorder;Acute Undifferentiated Leukemia;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;Blastic Plasmacytoid Dendritic Cell Neoplasm;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Burkitt Lymphoma;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Cutaneous B-cell Non-Hodgkin Lymphoma;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Intraocular Lymphoma;Juvenile Myelomonocytic Leukemia;Mast Cell Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncutaneous Extranodal Lymphoma;Peripheral T-cell Lymphoma;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Systemic Amyloidosis;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 Hairy Cell Leukemia;Refractory Multiple Myeloma;Small Intestine Lymphoma;Splenic Marginal Zone Lymphoma;Stage II Multiple Myeloma;Stage III Multiple Myeloma;T-cell Large Granular Lymphocyte Leukemia;Testicular Lymphoma;Untreated Adult Acute Lymphoblastic Leukemia;Untreated Adult Acute Myeloid Leukemia;Untreated Childhood Acute Lymphoblastic Leukemia;Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies;Waldenström MacroglobulinemiaDrug: fludarabine phosphate;Radiation: total-body irradiation;Procedure: peripheral blood stem cell transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Drug: cyclosporine;Drug: mycophenolate mofetil;Other: laboratory biomarker analysisFred Hutchinson Cancer Research CenterNational Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI)CompletedN/A74 YearsAll160N/AUnited States;Germany;Italy
2NCT00006251
(ClinicalTrials.gov)
May 200011/9/2000Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine, Mycophenolate Mofetil, Donor Lymphocyte Infusion in Treating Patients With Hematopoietic CancerInduction of Mixed Hematopoietic Chimerism in Patients Using Fludarabine, Low Dose TBI, PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate MofetilAcute Undifferentiated Leukemia;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Childhood Burkitt Lymphoma;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Cutaneous B-cell Non-Hodgkin Lymphoma;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Intraocular Lymphoma;Juvenile Myelomonocytic Leukemia;Mast Cell Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Myeloid/NK-cell Acute Leukemia;Nodal Marginal Zone B-cell Lymphoma;Noncutaneous Extranodal Lymphoma;Peripheral T-cell Lymphoma;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Systemic Amyloidosis;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 Renal Cell Cancer;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Small Intestine Lymphoma;Splenic Marginal Zone Lymphoma;Stage II Multiple Myeloma;Stage III Multiple Myeloma;T-cell Large Granular Lymphocyte Leukemia;Testicular Lymphoma;Waldenström MacroglobulinemiaRadiation: total-body irradiation;Drug: fludarabine phosphate;Drug: cyclosporine;Drug: mycophenolate mofetil;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation;Biological: donor lymphocytes;Procedure: peripheral blood stem cell transplantation;Other: laboratory biomarker analysisFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)CompletedN/A74 YearsAll21Phase 1/Phase 2United States;Italy;Germany
3NCT00112593
(ClinicalTrials.gov)
November 19992/6/2005Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without CancerAllogeneic Hematopoietic Stem Cell Transplantation for Induction of Mixed Hematopoietic Chimerism in Patients Infected With Human Immunodeficiency Virus-1 Using a Non-Marrow Ablative Conditioning Regimen Containing Total Body Irradiation in Combination With Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate MofetilAccelerated Phase Chronic Myelogenous Leukemia;Acute Undifferentiated Leukemia;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 Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Aggressive NK-cell Leukemia;AIDS-related Diffuse Large Cell Lymphoma;AIDS-related Diffuse Mixed Cell Lymphoma;AIDS-related Diffuse Small Cleaved Cell Lymphoma;AIDS-related Immunoblastic Large Cell Lymphoma;AIDS-related Lymphoblastic Lymphoma;AIDS-related Peripheral/Systemic Lymphoma;AIDS-related Primary CNS Lymphoma;AIDS-related Small Noncleaved Cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Blastic Phase Chronic Myelogenous Leukemia;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Burkitt Lymphoma;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;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;Contiguous Stage II Adult Burkitt Lymphoma;Contiguous Stage II Adult Diffuse Large Cell Lymphoma;Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Contiguous Stage II Adult Lymphoblastic Lymphoma;Contiguous Stage II Grade 1 Follicular Lymphoma;Contiguous Stage II Grade 2 Follicular Lymphoma;Contiguous Stage II Grade 3 Follicular Lymphoma;Contiguous Stage II Mantle Cell Lymphoma;Contiguous Stage II Marginal Zone Lymphoma;Contiguous Stage II Small Lymphocytic Lymphoma;Cutaneous B-cell Non-Hodgkin Lymphoma;Essential Thrombocythemia;Extramedullary Plasmacytoma;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;HIV Infection;HIV-associated Hodgkin Lymphoma;Intraocular Lymphoma;Isolated Plasmacytoma of Bone;Juvenile Myelomonocytic Leukemia;Mast Cell Leukemia;Meningeal Chronic Myelogenous Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Myeloid/NK-cell Acute Leukemia;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;Noncutaneous Extranodal Lymphoma;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Central Nervous System Lymphoma;Primary Myelofibrosis;Primary Systemic Amyloidosis;Progressive Hairy Cell Leukemia, Initial Treatment;Prolymphocytic Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Small Intestine Lymphoma;Splenic Marginal Zone Lymphoma;Stage 0 Chronic Lymphocytic Leukemia;Stage I Adult Burkitt Lymphoma;Stage I Adult Diffuse Large Cell Lymphoma;Stage I Adult Diffuse Mixed Cell Lymphoma;Stage I Adult Diffuse Small Cleaved Cell Lymphoma;Stage I Adult Hodgkin Lymphoma;Stage I Adult Immunoblastic Large Cell Lymphoma;Stage I Adult Lymphoblastic Lymphoma;Stage I Adult T-cell Leukemia/Lymphoma;Stage I Childhood Anaplastic Large Cell Lymphoma;Stage I Childhood Hodgkin Lymphoma;Stage I Childhood Large Cell Lymphoma;Stage I Childhood Lymphoblastic Lymphoma;Stage I Childhood Small Noncleaved Cell Lymphoma;Stage I Chronic Lymphocytic Leukemia;Stage I Cutaneous T-cell Non-Hodgkin Lymphoma;Stage I Grade 1 Follicular Lymphoma;Stage I Grade 2 Follicular Lymphoma;Stage I Grade 3 Follicular Lymphoma;Stage I Mantle Cell Lymphoma;Stage I Marginal Zone Lymphoma;Stage I Multiple Myeloma;Stage I Small Lymphocytic Lymphoma;Stage IA Mycosis Fungoides/Sezary Syndrome;Stage IB Mycosis Fungoides/Sezary Syndrome;Stage II Adult Hodgkin Lymphoma;Stage II Adult T-cell Leukemia/Lymphoma;Stage II Childhood Anaplastic Large Cell Lymphoma;Stage II Childhood Hodgkin Lymphoma;Stage II Childhood Large Cell Lymphoma;Stage II Childhood Lymphoblastic Lymphoma;Stage II Childhood Small Noncleaved Cell Lymphoma;Stage II Chronic Lymphocytic Leukemia;Stage II Cutaneous T-cell Non-Hodgkin Lymphoma;Stage II Multiple Myeloma;Stage IIA Mycosis Fungoides/Sezary Syndrome;Stage IIB Mycosis Fungoides/Sezary Syndrome;Stage III Adult Burkitt Lymphoma;Stage III Adult Diffuse Large Cell Lymphoma;Stage III Adult Diffuse Mixed Cell Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Hodgkin Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Adult T-cell Leukemia/Lymphoma;Stage III Childhood Anaplastic Large Cell Lymphoma;Stage III Childhood Hodgkin Lymphoma;Stage III Childhood Large Cell Lymphoma;Stage III Childhood Lymphoblastic Lymphoma;Stage III Childhood Small Noncleaved Cell Lymphoma;Stage III Chronic Lymphocytic Leukemia;Stage III Cutaneous T-cell Non-Hodgkin 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 Multiple Myeloma;Stage III Small Lymphocytic Lymphoma;Stage IIIA Mycosis Fungoides/Sezary Syndrome;Stage IIIB Mycosis Fungoides/Sezary Syndrome;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Large Cell Lymphoma;Stage IV Adult Diffuse Mixed Cell Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Hodgkin Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Adult T-cell Leukemia/Lymphoma;Stage IV Childhood Anaplastic Large Cell Lymphoma;Stage IV Childhood Hodgkin Lymphoma;Stage IV Childhood Large Cell Lymphoma;Stage IV Childhood Lymphoblastic Lymphoma;Stage IV Childhood Small Noncleaved Cell Lymphoma;Stage IV Chronic Lymphocytic Leukemia;Stage IV Cutaneous T-cell Non-Hodgkin 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;Stage IVA Mycosis Fungoides/Sezary Syndrome;Stage IVB Mycosis Fungoides/Sezary Syndrome;T-cell Large Granular Lymphocyte Leukemia;Testicular Lymphoma;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol Specific;Waldenström MacroglobulinemiaDrug: fludarabine phosphate;Radiation: total-body irradiation;Procedure: peripheral blood stem cell transplantation;Drug: cyclosporine;Drug: mycophenolate mofetil;Other: laboratory biomarker analysisFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)CompletedN/A75 YearsAll5N/AUnited States

36. 表皮水疱症


臨床試験数 : 163 薬物数 : 185 - (DrugBank : 46) / 標的遺伝子数 : 50 - 標的パスウェイ数 : 125
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02582775
(ClinicalTrials.gov)
March 201616/10/2015MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCsMT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell InfusionsEpidermolysis BullosaProcedure: Bone marrow infusion;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Biological: Donor mesenchymal stem cell infusions;Drug: Busulfan;Drug: Thymoglobulin;Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total Body IrradiationMasonic Cancer Center, University of MinnesotaNULLActive, not recruitingN/A25 YearsAll17Phase 2United States
2EUCTR2012-000605-72-NL
(EUCTR)
06/02/201412/08/2013Stem cell transplantation with cord blood and mesenchymal stem cells after reduced intensity conditioning for severe forms of the blistering disease epidermolysis bullosaUnrelated cord blood transplantation after reduced toxicity conditioning with mesenchymal stromal cell co-infusion in patients with severe epidermolysis bullosa - CB+MSCforEB The source population consists of patients referred to or within the UMC Groningen because they have diagnosed clinically and genetically severe generalized RDEB.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: TC-MSC
INN or Proposed INN: Mesenchymal stromal cells
Trade Name: Busilvex
Trade Name: Fludarabine
Product Name: FLudarabine
Trade Name: THYMOGLOBULINE
Product Name: Thymoglobuline
Universitair Medisch Centrum UtrechtNULLNot RecruitingFemale: yes
Male: yes
11Phase 2Netherlands
3NCT01033552
(ClinicalTrials.gov)
January 201014/12/2009Biochemical Correction of Severe EB by Allo HSCT and Off-the-shelf MSCsMT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem CellsEpidermolysis BullosaDrug: Cyclophosphamide;Drug: Fludarabine;Drug: Anti-thymocyte globulin;Drug: Cyclosporine A;Drug: Mycophenolate mofetil;Procedure: Mesenchymal stem cell transplantation;Radiation: Total body irradiation;Procedure: Bone marrow or umbilical cord blood (UCG) stem cell transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A25 YearsAll32Phase 1/Phase 2United States
4NCT00881556
(ClinicalTrials.gov)
August 20, 200914/4/2009Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB)Epidermolysis BullosaDrug: Palifermin;Drug: Fludarabine;Drug: Busulfan;Drug: Lorazepam;Drug: Alemtuzumab;Drug: TacrolimusColumbia UniversityNULLTerminatedN/A21 YearsAll3Early Phase 1United States
5NCT00478244
(ClinicalTrials.gov)
April 200723/5/2007Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis BullosaAllogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis BullosaEpidermolysis BullosaDrug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: hematopoietic bone marrow transplantationMasonic Cancer Center, University of MinnesotaNULLTerminatedN/A25 YearsAll7N/AUnited States

46. 悪性関節リウマチ


臨床試験数 : 4,356 薬物数 : 2,567 - (DrugBank : 415) / 標的遺伝子数 : 192 - 標的パスウェイ数 : 228
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00001677
(ClinicalTrials.gov)
June 19983/11/1999Methotrexate Alone Versus Combination of Methotrexate and Subcutaneous Fludarabine for Severe Rheumatoid Arthritis: Safety, Tolerance and EfficacyMethotrexate Alone Versus Combination of Methotrexate and Subcutaneous Fludarabine for Severe Rheumatoid Arthritis: Safety, Tolerance and EfficacyArthritis, Rheumatoid;SynovitisDrug: Methotrexate;Drug: FludarabineNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NULLCompletedN/AN/ABoth40Phase 2United States

49. 全身性エリテマトーデス


臨床試験数 : 993 薬物数 : 702 - (DrugBank : 184) / 標的遺伝子数 : 116 - 標的パスウェイ数 : 200
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT02080195
(ClinicalTrials.gov)
September 13, 201628/2/2014Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE)A Phase I/II Study of Nonmyeloablative Conditioning and Transplantation of Human Leukocyte Antigen (HLA)-Matched, Partially HLA-mismatched, HLA-haploidentical or Matched Unrelated Bone Marrow for Patients With Refractory SLELupus Erythematosus;Graft-versus-host DiseaseDrug: Cyclophosphamide;Drug: Fludarabine;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Drug: Rabbit antithymocyte globulin;Radiation: Total body irradiation;Biological: Allogeneic bone marrow transplantSidney Kimmel Comprehensive Cancer Center at Johns HopkinsMaryland Stem Cell Research FundTerminated18 Years75 YearsAll1Phase 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
3NCT00076752
(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
4NCT00001676
(ClinicalTrials.gov)
January 19983/11/1999Cyclophosphamide and Fludarabine to Treat Lupus NephritisCombination of Cyclophosphamide and Fludarabine for Lupus Nephritis: Tolerance, Toxicity, Efficacy and Effects on B and T Lymphocyte RegenerationGlomerulonephritis;Lupus Nephritis;Systemic Lupus ErythematosusDrug: SQ FludarabineNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NULLCompletedN/AN/ABoth15Phase 1United States

51. 全身性強皮症


臨床試験数 : 525 薬物数 : 565 - (DrugBank : 148) / 標的遺伝子数 : 114 - 標的パスウェイ数 : 217
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03593902
(ClinicalTrials.gov)
May 17, 201828/6/2018Cardiac Safe Transplants for Systemic SclerosisAutologous Hematopoietic Stem Cell Transplant for Patients With Systemic Sclerosis and Cardiac DysfunctionSystemic Sclerosis;SclerodermaDrug: Rituximab;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Mesna;Drug: rATG;Drug: Methylprednisolone;Drug: G-CSF;Biological: IVIg;Biological: Autologous Stem CellsNorthwestern UniversityNULLTerminated18 Years65 YearsAll9Phase 2/Phase 3United States
2NCT01445821
(ClinicalTrials.gov)
September 15, 201129/9/2011Autologous Stem Cell Systemic Sclerosis Immune Suppression TrialRandomized Study of Different Non-myeloablative Conditioning Regimens With Hematopoietic Stem Cell Support in Patients With Scleroderma (Autologous Systemic Sclerosis Immune Suppression Trial - II ASSIST-IIb)Scleroderma, SystemicBiological: Peripheral Blood Stem Cells;Drug: Cyclophosphamide;Drug: Mesna;Drug: rATG;Drug: Methylprednisolone;Drug: Filgrastim;Drug: FludarabineNorthwestern UniversityNULLTerminated17 Years60 YearsAll44Phase 3United States
3NCT01047072
(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
4NCT00684255
(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
5NCT00622895
(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
6NCT00040651
(ClinicalTrials.gov)
July 20025/7/2002Safety and Value of Self Bone Marrow Transplants Following Chemotherapy in Scleroderma PatientsTransplantation With T-Cell Depleted Autologous Peripheral Stem Cells for Severe Systemic Sclerosis: A Phase I Dose Escalation StudyScleroderma;Systemic SclerosisDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Thymoglobulin;Procedure: Leukapheresis;Procedure: Self bone marrow transplantNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)University of Pittsburgh;Amgen;Genzyme, a Sanofi CompanyTerminated18 Years70 YearsBoth15Phase 1United States

60. 再生不良性貧血


臨床試験数 : 245 薬物数 : 318 - (DrugBank : 86) / 標的遺伝子数 : 44 - 標的パスウェイ数 : 166
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT05600426
(ClinicalTrials.gov)
January 25, 202320/10/2022A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation With Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)Severe Aplastic AnemiaDrug: cyclosporine;Procedure: Matched Unrelated Donor Hematopoetic Stem Cell Transplant;Drug: horse anti-thymocyte globulin (ATG);Drug: rabbit anti-thymocyte globulin (ATG);Drug: Methotrexate;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: low-dose total body irradiation (TBI);Procedure: Immunosuppressive Therapy (IST)Boston Children's HospitalCenter for International Blood and Marrow Transplant Research;National Institutes of Health (NIH);National Heart, Lung, and Blood Institute (NHLBI);North American Pediatric Aplastic Anemia Consortium;Pediatric Transplantation and Cellular Therapy Consortium;Blood and Marrow Transplant Clinical Trials NetworkRecruiting0 Years25 YearsAll234Phase 3United States
2NCT05031897
(ClinicalTrials.gov)
October 25, 202131/8/2021Reduced-Intensity Conditioning for the Prevention of Treatment-Related Mortality in Patients Who Undergo a Hematopoietic Stem Cell TransplantA 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity ConditioningAcute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Aplastic Anemia;Chronic Lymphocytic Leukemia;Chronic Myelomonocytic Leukemia;Essential Thrombocythemia;Hematopoietic and Lymphoid Cell Neoplasm;Hodgkin Lymphoma;Myelodysplastic Syndromes;Myelofibrosis;Myeloid Leukemia;Myeloid Neoplasm;Non-Hodgkin Lymphoma;Plasma Cell Myeloma;Polycythemia Vera;Small Lymphocytic LymphomaProcedure: Hematopoietic Cell Transplantation;Drug: Mycophenolate Mofetil;Drug: Tacrolimus;Drug: Cyclophosphamide;Radiation: Total-Body Irradiation;Procedure: Donor Lymphocyte Infusion;Drug: Fludarabine;Drug: MelphalanThomas Jefferson UniversityNULLRecruiting18 YearsN/AAll67Phase 2United States
3NCT03622788
(ClinicalTrials.gov)
August 8, 20193/8/2018Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell TransplantAnti-Viral Central Memory CD8 Veto Cells in Haploidentical Hematopoietic Stem Cell TransplantationFollicular Lymphoma;Hodgkin Lymphoma;Mantle Cell Lymphoma;Myelodysplastic Syndrome;Myeloproliferative Neoplasm;Non-Hodgkin Lymphoma;Plasma Cell Myeloma;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Aplastic Anemia;Bone Marrow Failure;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 PositiveBiological: Anti-Thymocyte Globulin;Drug: Cyclophosphamide;Biological: Cytokine-treated Veto Cells;Drug: Fludarabine;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationM.D. Anderson Cancer CenterNULLRecruiting12 Years75 YearsAll24Phase 1/Phase 2United States
4NCT03821987
(ClinicalTrials.gov)
December 17, 201823/1/2019Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAARisk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAAAplastic Anemia;Stem Cell Transplant Complications;Engraft FailureDrug: FludarabinePeking University People's HospitalNULLRecruiting3 Years55 YearsAll55N/AChina
5NCT03579875
(ClinicalTrials.gov)
November 13, 201825/5/2018T Cell Receptor a/ß TCD HCT in Patients With Fanconi AnemiaT Cell Receptor Alpha/Beta T Cell Depleted (a/ß TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA)Fanconi Anemia;Severe Aplastic Anemia;Myelodysplastic SyndromesDrug: Total Body Irradiation (TBI) (Plan 1);Drug: Cyclophosphamide (CY) (Plan 1);Drug: Fludarabine (FLU);Drug: Methylprednisolone (MP);Device: Donor mobilized PBSC infusion;Drug: G-CSF;Drug: Cyclophosphamide (CY) (Plan 2);Drug: Rituximab;Drug: BusulfanMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A65 YearsAll48Phase 2United States
6NCT03531736
(ClinicalTrials.gov)
May 9, 20189/5/2018T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic AnemiaAllogeneic Hematopoietic Stem Cell Transplantation of a/ß T-Lymphocyte Depleted Graft Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic AnemiaMyeloid DiseasesDrug: Antithymocyte globulin (Rabbit);Drug: fludarabine;Radiation: total body irradiation;Drug: cyclophosphamide;Drug: Rituxan;Procedure: Allogeneic Hematopoietic Stem Cell TransplantationMemorial Sloan Kettering Cancer CenterNULLRecruiting18 YearsN/AAll15Phase 1United States
7NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNULLActive, not recruiting1 Year75 YearsAll31Phase 2United States
8NCT03192397
(ClinicalTrials.gov)
August 9, 201716/6/2017Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell TransplantA Phase Ib/2 Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell TransplantationAcute Myeloid Leukemia in Remission;Adult Acute Lymphoblastic Leukemia in Complete Remission;Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission;Chronic Myelomonocytic Leukemia in Remission;Graft Versus Host Disease;Hodgkin Lymphoma;Minimal Residual Disease;Myelodysplastic Syndrome;Myeloproliferative Neoplasm;Non-Hodgkin Lymphoma;Plasma Cell Myeloma;Severe Aplastic Anemia;Waldenstrom MacroglobulinemiaProcedure: Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Melphalan Hydrochloride;Drug: Mycophenolate Mofetil;Drug: Sirolimus;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNULLActive, not recruiting18 YearsN/AAll35Phase 1/Phase 2United States
9NCT02918292
(ClinicalTrials.gov)
July 3, 201727/9/2016Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)Optimizing Haploidentical Aplastic Anemia Transplantation (CHAMP) (BMT CTN 1502)Severe Aplastic AnemiaDrug: Antithymocyte Globulin (ATG);Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation (TBI);Procedure: Haplo HSCT;Drug: Tacrolimus;Drug: Mycophenolate mofetil (MMF);Drug: G-CSFMedical College of WisconsinNational Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI);Blood and Marrow Transplant Clinical Trials Network;National Marrow Donor ProgramCompletedN/A75 YearsAll32Phase 2United States
10NCT02960646
(ClinicalTrials.gov)
January 18, 20178/11/2016Engineered Donor Stem Cell Transplant in Treating Patients With Hematologic MalignanciesPhase I Clinical Trial Using an Engineered Peripheral Blood Graft for Haploidentical TransplantationAcute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome;Aplastic Anemia;Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Lymphoblastic Lymphoma;Myelodysplastic Syndrome;Myeloproliferative Neoplasm;Plasma Cell Myeloma;Recurrent Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma;Recurrent Hodgkin Lymphoma;Recurrent Non-Hodgkin Lymphoma;Recurrent Plasma Cell Myeloma;Therapy-Related Myelodysplastic SyndromeDrug: Cyclophosphamide;Biological: Filgrastim;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Melphalan;Procedure: Peripheral Blood Stem Cell Transplantation;Biological: Rituximab;Drug: Tacrolimus;Radiation: Total-Body IrradiationM.D. Anderson Cancer CenterNational Cancer Institute (NCI)Completed18 Years65 YearsAll11Phase 1United States
11ChiCTR1900023509
2017-01-012019-05-31Allogeneic Hematopoietic Stem Cell Transplantation with Risk-adapted Conditioning Regimens for Children with Aplastic Anemia or Refractory Cytopenia of Childhood: a Single-Center Prospective TrialAllogeneic Hematopoietic Stem Cell Transplantation with Risk-adapted Conditioning Regimens for Children with Aplastic Anemia or Refractory Cytopenia of Childhood: a Single-Center Prospective Trial aplastic anemia,Refractory Cytopenia of Childhood1:Fludarabine150 mg/m2 +Cyclophosphamide200 mg/kg+ Thymoglobuline10mg/kg;2:Fludarabine200 mg/m2 +Cyclophosphamide120 mg/kg+ Thymoglobuline10mg/kg+300cGy TBI;3:Fludarabine200 mg/m2 +Cyclophosphamide120 mg/kg+ Thymoglobuline10mg/kg+ Busulfan 8mg/kg;Shanghai Children's Medical Center affiliated to Shanghai Jiao Tong University School of MedicineNULLRecruiting18Both1:50;2:50;3:50;China
12NCT02828592
(ClinicalTrials.gov)
September 9, 20166/7/2016Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic AnemiaA Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related DonorSevere Aplastic AnemiaDrug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Drug: Rabbit ATGNorthside Hospital, Inc.NULLRecruiting1 Year75 YearsAll20Phase 2United States
13NCT02833805
(ClinicalTrials.gov)
September 201612/7/2016NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic AnemiaA Phase II Trial of Non-Myeloablative (NMA) Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Donor (MUD) Bone Marrow for Newly Diagnosed Patients With Severe Aplastic AnemiaSevere Aplastic Anemia;Aplastic Anemia;Bone Marrow Failure;ImmunosuppressionDrug: Thymoglobulin;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total body irradiation;Drug: Tacrolimus;Drug: Mycophenolate mofetilSidney Kimmel Comprehensive Cancer Center at Johns HopkinsNULLCompletedN/AN/AAll21Phase 2United States
14NCT02845596
(ClinicalTrials.gov)
August 20169/5/2016Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic AnemiaUnrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic AnemiaSevere Aplastic AnemiaDrug: cyclosporine;Procedure: Matched Unrelated Donor Hematopoietic Stem Cell Transplant;Drug: horse anti-thymocyte globulin (ATG);Drug: rabbit anti-thymocyte globulin (ATG);Drug: methotrexate;Drug: fludarabine;Drug: cyclophosphamide;Radiation: low-dose total body irradiation (TBI);Procedure: Immunosuppressive Therapy (IST)Michael Pulsipher, MDNULLActive, not recruitingN/A25 YearsAll40N/AUnited States
15NCT02566304
(ClinicalTrials.gov)
November 13, 201530/9/2015Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Two Step Approach to Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic MalignanciesAcute Myeloid Leukemia;Acute Myeloid Leukemia in Remission;Aplastic Anemia;Chronic Myelomonocytic Leukemia;Hodgkin Lymphoma;Indolent Non-Hodgkin Lymphoma;Malignant Neoplasm;Myelodysplastic Syndrome;Myeloproliferative Neoplasm;Plasma Cell Myeloma;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring SideroblastsDrug: Fludarabine;Radiation: Total-Body Irradiation;Biological: T Cell-Depleted Donor Lymphocyte Infusion;Drug: Cyclophosphamide;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Tacrolimus;Drug: Mycophenolate mofetil;Other: Laboratory Biomarker AnalysisSidney Kimmel Cancer Center at Thomas Jefferson UniversityNULLRecruiting18 YearsN/AAll35Phase 2United States
16NCT02162420
(ClinicalTrials.gov)
January 10, 201510/6/2014Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaHematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLRecruiting0 Years70 YearsAll50N/AUnited States
17NCT02224872
(ClinicalTrials.gov)
August 201418/8/2014Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic AnemiaA Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia and Other Bone Marrow Failure SyndromesSevere Aplastic Anemia;Bone Marrow Failure SyndromesProcedure: Bone marrow transplant;Drug: Thymoglobulin;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: TBI;Drug: Mesna;Drug: Tacrolimus;Drug: Mycophenolic acid mofetilSidney Kimmel Comprehensive Cancer Center at Johns HopkinsNULLCompletedN/A73 YearsAll18Phase 2United States
18NCT01760655
(ClinicalTrials.gov)
December 24, 20122/1/2013Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in Treating Patients With High-Risk Hematologic MalignanciesA Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Hematologic MalignanciesAcute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Acute Myeloid Leukemia With Inv(3) (q21.3;q26.2); GATA2, MECOM;Acute Myeloid Leukemia With t(6;9) (p23;q34.1); DEK-NUP214;Aplastic Anemia;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Follicular Lymphoma;Hodgkin Lymphoma;Myelodysplastic Syndrome;Myelofibrosis;Myeloid Leukemia;Non-Hodgkin Lymphoma;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Adult Acute Myeloid Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Refractory Acute Myeloid Leukemia;Secondary Acute Myeloid Leukemia;Therapy-Related Acute Myeloid LeukemiaDrug: Fludarabine phosphate;Drug: Busulfan;Radiation: Total-Body Irradiation;Biological: Therapeutic Allogeneic Lymphocytes;Drug: Cyclophosphamide;Procedure: Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Other: Laboratory Biomarker AnalysisSidney Kimmel Cancer Center at Thomas Jefferson UniversityNational Cancer Institute (NCI)CompletedN/AN/AAll62Phase 2United States
19NCT01759732
(ClinicalTrials.gov)
September 201230/12/2012Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic AnemiaHaploidentical Stem Cell Transplantation With Fixed Dose of T Cells After in Vitro T Cell Depletion Using CD3 Monoclonal Antibody for Children With Acquired Severe Aplastic AnemiaAcquired Aplastic AnemiaDrug: Fludarabine;Drug: Cyclophosphamide;Biological: anti-thymocyte globulin;Biological: filgrastim;Radiation: Total body irradiation;Procedure: CD3-depleted hematopoietic cell transplantationAsan Medical CenterNULLRecruitingN/A21 YearsBoth10Phase 2Korea, Republic of
20NCT01659606
(ClinicalTrials.gov)
July 20126/8/2012Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaRadiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere DiseaseDyskeratosis Congenita;Hoyeraal Hreidarsson Syndrome;Revesz Syndrome;Aplastic AnemiaBiological: alemtuzumab;Drug: Fludarabine;Drug: Cyclosporins;Drug: Mycophenolate mofetil;Drug: TacrolimusBoston Children's HospitalDana-Farber Cancer Institute;Children's Hospital Medical Center, Cincinnati;Children's Hospital Los Angeles;Fred Hutch/University of Washington/Seattle Children's Cancer Consortium;Baylor College of Medicine;Children's Hospital of Philadelphia;University of Wisconsin, Madison;Karolinska University Hospital;Hackensack Meridian Health;Duke University;Oslo University Hospital;Children's Mercy Hospital Kansas City;Mayo Clinic;University of Chicago;Massachusetts General HospitalActive, not recruiting30 Days65 YearsAll40Phase 2United States;Norway;Sweden
21NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;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 Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;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 Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
22NCT01500161
(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
23NCT01472055
(ClinicalTrials.gov)
October 20116/11/2011Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell TransplantationPharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell TransplantationAcute Leukemia;Chronic Leukemia;Severe Aplastic AnemiaDrug: FludarabineSeoul National University HospitalMinistry of Food and Drug Safety, KoreaRecruitingN/A19 YearsBoth46Phase 2Korea, Republic of
24NCT01384513
(ClinicalTrials.gov)
August 4, 201127/6/2011A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological MalignanciesA Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic MalignanciesAdult 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;Aplastic Anemia;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Myelodysplastic Syndromes;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Essential Thrombocythemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Juvenile Myelomonocytic Leukemia;Mastocytosis;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Polycythemia Vera;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Anemia;Refractory Anemia With Ringed Sideroblasts;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenström Macroglobulinemia;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)Drug: Fludarabine;Drug: Busulfan;Radiation: Total Body Irradiation (TBI);Biological: Donor Lymphocyte Infusion (DLI);Drug: Cyclophosphamide (CY);Drug: Tacrolimus;Drug: Mycophenolate mofetil;Device: Allogeneic hematopoietic stem cell transplantation;Procedure: Peripheral blood stem cell transplantation (PBSCT)Sidney Kimmel Cancer Center at Thomas Jefferson UniversityNULLCompleted18 YearsN/AAll40Phase 2United States
25JPRN-UMIN000006071
2011/08/0131/08/2011Allogeneic hematopoietic stem cell transplantation for aplastic anemia using low-dose anti-thymocyte globulinAllogeneic hematopoietic stem cell transplantation for aplastic anemia using low-dose anti-thymocyte globulin - HSCT for AA using low-dose ATG SAAConditioning regimen
Fludarabine
30mg/m2/day iv day-6,-5,-4,-3
Cyclophosphamide
25mg/kg/day iv day-6,-5,-4,-3
ATG(thymoglobulin)
1.25mg/kg iv day-4,-3

In case of HSCT from unrelated donor or HLA-mismatched related donor,
TBI 2Gy day-1 will be added.
Kanto Study Group for Cell TherapyNULLComplete: follow-up complete16years-old65years-oldMale and Female28Phase 2Japan
26NCT01187017
(ClinicalTrials.gov)
August 201020/8/2010A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic AnemiaA Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic AnemiaAplastic Anemia;Neutropenia;Pancytopenia;Severe Aplastic AnemiaDrug: Cyclophosphamide;Drug: FludarabineNational Heart, Lung, and Blood Institute (NHLBI)NULLCompleted2 YearsN/AAll1Phase 1/Phase 2United States
27NCT01145976
(ClinicalTrials.gov)
March 201019/5/2010Comparison of Cy-Atg vs Flu-Atg for the Conditioning Therapy in Allo-HCT for Adult Aplastic AnemiaRandomized Comparison of Cyclophosphamide Versus Fludarabine in Addition to Anti-thymocyte Globulin for the Conditioning Therapy in Allogeneic Hematopoietic Cell Transplantation for Adult Acquired Aplastic AnemiaAplastic AnemiaDrug: Cy-ATG;Drug: Flu-ATGCooperative Study Group A for HematologyNULLRecruiting15 Years65 YearsBoth98Phase 3Korea, Republic of
28NCT01129323
(ClinicalTrials.gov)
November 200921/4/2010Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic AnemiaReduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic AnemiaSevere Aplastic AnemiaDrug: Cyclophosphamide, Fludarabine, Rabbit ATGCity of Hope Medical CenterNULLWithdrawnN/A21 YearsBoth0N/AUnited States
29NCT00987480
(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
30NCT01105273
(ClinicalTrials.gov)
July 20098/4/2010Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic AnemiaHLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte GlobulinAplastic AnemiaBiological: anti-thymocyte globulin;Biological: filgrastim;Drug: Fludarabine;Drug: Cyclophosphamide;Procedure: CD3±CD19 depleted hematopoietic stem cell transplantationAsan Medical CenterNULLCompletedN/A21 YearsBoth12Phase 1/Phase 2Korea, Republic of
31NCT00856388
(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
32NCT00882323
(ClinicalTrials.gov)
November 200815/4/2009Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic AnemiaAplastic AnemiaDrug: Cyclophosphamide, Fludarabine, ThymoglobulinThe Korean Society of Pediatric Hematology OncologyNULLRecruiting1 Year21 YearsBoth33Phase 2Korea, Republic of
33EUCTR2007-001657-26-GB
(EUCTR)
29/02/200807/11/2007Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies.Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies. 1.Acute, chronic leukaemia or myelodysplastic syndrome for which allogeneic transplantation is considered as the best treatment option. 2.Acute lymphoblastic leukaemia (ALL)3.Non-Hodgkin’s lymphoma4.Hodgkin’s disease5.Chronic lymphocytic leukaemia. 6.Acquired bone marrow failure syndromes7.Other haematological malignancies for which UD bone marrow transplantation is indicated
MedDRA version: 9.1;Level: LLT;Classification code 10000880;Term: Acute myeloid leukaemia
MedDRA version: 9.1;Classification code 10028533;Term: Myelodysplastic syndrome
MedDRA version: 9.1;Classification code 10009013;Term: Chronic myeloid leukaemia
MedDRA version: 9.1;Classification code 10000844;Term: Acute lymphoblastic leukaemia
MedDRA version: 9.1;Classification code 10020328;Term: Hodgkin's lymphoma
MedDRA version: 9.1;Classification code 10029593;Term: Non-Hodgkin's lymphoma NOS
MedDRA version: 9.1;Classification code 10003892;Term: B-cell chronic lymphocytic leukaemia/prolymphocytic leukaemia/small lymphocytic lymphoma
MedDRA version: 9.1;Classification code 10002968;Term: Aplastic anaemia, unspecified
Trade Name: Fludarabine
INN or Proposed INN: FLUDARABINE PHOSPHATE
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
Trade Name: Thymoglobulin
Other descriptive name: RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN
Trade Name: Thiotepa
INN or Proposed INN: THIOTEPA
Trade Name: Cyclophosphamide
Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE
Trade Name: Alkeran
INN or Proposed INN: MELPHALAN
King's College Hospital NHS Foundation TrustNULLNot RecruitingFemale: yes
Male: yes
27Phase 2United Kingdom
34NCT00455312
(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
35NCT00358657
(ClinicalTrials.gov)
May 24, 200628/7/2006Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited DisordersHLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States
36NCT00474747
(ClinicalTrials.gov)
February 7, 200616/5/2007Fludarabine-Based Conditioning for Allogeneic Marrow Transplantation in Aplastic AnemiaFludarabine-Based Conditioning for Allogeneic Marrow Transplantation From HLA-Compatible Unrelated Donors in Severe Aplastic AnemiaAplastic AnemiaDrug: Antithymocyte Globulin;Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total Body Irradiation (TBI);Procedure: Bone Marrow TransplantM.D. Anderson Cancer CenterNational Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI)CompletedN/A65 YearsAll5Phase 1/Phase 2United States
37NCT00326417
(ClinicalTrials.gov)
January 200612/5/2006Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)Fludarabine-based Conditioning for Allogeneic Marrow Transplantation From HLA-compatible Unrelated Donors in Severe Aplastic Anemia (BMT CTN #0301)Anemia, AplasticDrug: Fludarabine;Drug: Cyclophosphamide 150mg;Drug: Cyclophosphamide 100mg;Drug: Cyclophosphamide 50mgMedical College of WisconsinNational Heart, Lung, and Blood Institute (NHLBI);Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor ProgramCompletedN/A65 YearsAll97Phase 1/Phase 2United States
38NCT00737685
(ClinicalTrials.gov)
January 200618/8/2008Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic AnemiaAnemia, AplasticDrug: cyclophosphamide, fludarabine , thymoglobulinThe Korean Society of Pediatric Hematology OncologyNULLActive, not recruitingN/A25 YearsBoth30Phase 2Korea, Republic of
39NCT00295997
(ClinicalTrials.gov)
May 200523/2/2006Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer, Metastatic Kidney Cancer, or Aplastic AnemiaNon-myeloablative Allogeneic Stem Cell Transplantation With Match Unrelated Donors for Treatment of Hematologic Malignancies and Renal Cell Carcinoma and Aplastic AnemiaChronic Myeloproliferative Disorders;Kidney Cancer;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative NeoplasmsBiological: anti-thymocyte globulin;Biological: filgrastim;Biological: graft-versus-tumor induction therapy;Biological: therapeutic allogeneic lymphocytes;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methotrexate;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)Active, not recruitingN/A74 YearsBoth35N/AUnited States
40JPRN-C000000356
2004/12/0120/03/2006Evaluation of the safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) using alemtuzumab for patients with aplastic anemiaEvaluation of the safety and efficacy of allogeneic hematopoietic stem cell transplantation (HSCT) using alemtuzumab for patients with aplastic anemia - HSCT for aplastic anemia using alemtuzumab Aplastic anemiaAlemtuzumab is added to the fludarabine-based regimen at 0.16 – 0.25 mg/kg per day for 6 days (days -10 to -5).GCP-ISS HE0403 groupNULLComplete: follow-up complete20years-old65years-oldMale and Female38Phase 1,2Japan
41NCT00533923
(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
42NCT00186797
(ClinicalTrials.gov)
December 20029/9/2005Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic AnemiaAplastic AnemiaProcedure: Allogeneic stem cell transplant;Drug: Fludarabine, CyclophosphamideSt. Jude Children's Research HospitalNULLCompletedN/A21 YearsBoth28N/AUnited States
43NCT00516152
(ClinicalTrials.gov)
November 200213/8/2007Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCTPhase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell TransplantationChronic Myeloid Leukemia;Acute Myelogenous Leukemia;Myelodysplasia;Acute Lymphocytic Leukemia;Severe Aplastic Anemia;Non-Hodgkin's Lymphoma;Lymphoproliferative Disease;Multiple Myeloma;Advanced Myeloproliferative DiseaseDrug: Busulfan/Fludarabine phosphate/Tacrolimus/Methotrexate/G-CSFUniversity of California, San FranciscoNULLCompleted15 Years61 YearsBoth36Phase 2United States
44NCT00054236
(ClinicalTrials.gov)
May 20025/2/2003Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic AnemiaPilot Study Of Multiple Umbilical Cord Blood Unit Transplantation Following Non-Myeloablative Conditioning In Patients With Hematologic Disorders Or Severe Aplastic AnemiaChronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative DiseasesBiological: anti-thymocyte globulin;Biological: filgrastim;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: umbilical cord blood transplantation;Drug: methylprednisoloneCase Comprehensive Cancer CenterNULLCompletedN/A120 YearsAll55Phase 1United States
45NCT00053989
(ClinicalTrials.gov)
January 29, 20025/2/2003NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/DisordersNon-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and DisordersChronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic AnemiaBiological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Biological: sargramostim;Biological: therapeutic allogeneic lymphocytes;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationRoswell Park Cancer InstituteNULLCompleted4 Years75 YearsAll41Phase 2United States
46NCT00590460
(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
47NCT00427336
(ClinicalTrials.gov)
December 200025/1/2007Purine Analog-Based Conditioning in Patients With Severe Aplastic AnemiaPurine Analog-Based Conditioning for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic AnemiaAplastic AnemiaDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Antithymocyte GlobulinM.D. Anderson Cancer CenterNULLCompletedN/A70 YearsAll9N/AUnited States
48NCT00006379
(ClinicalTrials.gov)
June 20004/10/2000Non-Ablative Allo HSCT For Hematologic Malignancies or SAAPurine-Analog-Containing Non-Myeloablative Allogeneic Stem Cell Transplantation for Treatment of Hematologic Malignancies and Severe Aplastic AnemiaChronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Precancerous/Nonmalignant Condition;Small Intestine CancerBiological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: peripheral blood stem cell transplantationCase Comprehensive Cancer CenterNational Cancer Institute (NCI)CompletedN/A70 YearsBoth58Phase 2United States
49NCT01499147
(ClinicalTrials.gov)
February 200023/11/2011Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic MalignanciesFludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic MalignanciesAcute Myeloid Leukemia;Acute Leukemia;Chronic Myelogenous Leukemia;Malignant Lymphoma;Hodgkin's Disease;Multiple Myeloma;Lymphocytic Leukemia;Myeloproliferative Disorder;Polycythemia Vera;Myelofibrosis;Aplastic AnemiaDrug: fludarabine/busulfan;Drug: fludarabine/ melphalan;Drug: ATGUniversity of Illinois at ChicagoNULLCompleted10 Years65 YearsAll100N/AUnited States
50NCT00636909
(ClinicalTrials.gov)
July 199910/3/2008Nonmyeloablative Allo SCT for the Treatment of Hematologic DisordersNonmyeloablative Allogeneic Stem Cell Transplant for the Treatment of Hematologic DisordersAML;ALL;CML Chronic Phase, Accelerated Phase, or Blast Crisis;CLL;MDS;RELAPSED NON-HODGKIN'S OR HODGKIN'S LYMPHOMA;APLASTIC ANEMIA;MULTIPLE MYELOMA;MYELOPROLIFERATIVE DISORDER (P Vera, CMML, ET)Drug: Cyclophosphamide;Drug: fludarabine;Drug: cyclosporine;Drug: methotrexate;Biological: G-CSFBeth Israel Deaconess Medical CenterAmgenCompletedN/A65 YearsAll25Phase 2NULL
51NCT00003816
(ClinicalTrials.gov)
October 19, 19981/11/1999Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic CancerAllogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic AnemiaChronic Myeloproliferative Disorders;Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Nonmalignant Neoplasm;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol SpecificBiological: anti-thymocyte globulin;Drug: busulfan;Drug: carboplatin;Drug: cyclophosphamide;Drug: etoposide;Drug: fludarabine phosphate;Drug: melphalan;Drug: thiotepa;Radiation: total-body irradiationRoswell Park Cancer InstituteNULLCompleted4 Years70 YearsAll362Phase 2/Phase 3United States

61. 自己免疫性溶血性貧血


臨床試験数 : 146 薬物数 : 131 - (DrugBank : 59) / 標的遺伝子数 : 28 - 標的パスウェイ数 : 158
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00373594
(ClinicalTrials.gov)
June 20057/9/2006Therapy for Chronic Cold Agglutinin DiseaseTherapy for Chronic Cold Agglutinin Disease: A Prospective, Non-randomized International Multicentre Study on the Safety and Efficacy of Rituximab in Combination With Fludarabine.Cold Agglutinin DiseaseDrug: Rituximab;Drug: FludarabineUniversity of BergenNULLCompleted18 Years90 YearsBoth30Phase 2Norway;Russian Federation

62. 発作性夜間ヘモグロビン尿症


臨床試験数 : 292 薬物数 : 151 - (DrugBank : 49) / 標的遺伝子数 : 22 - 標的パスウェイ数 : 108
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04965597
(ClinicalTrials.gov)
April 19, 202221/6/2021Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure DiseasesBone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond SyndromeDrug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life AssessmentFred Hutchinson Cancer CenterBlood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)Recruiting1 Year49 YearsAll40Phase 2United States
2NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNULLActive, not recruiting1 Year75 YearsAll31Phase 2United States
3NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;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 Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;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 Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
4NCT00856388
(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
5NCT00397813
(ClinicalTrials.gov)
January 20069/11/2006Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative DisordersLow-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Center TrialAtypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Chronic Myelomonocytic Leukemia;de Novo Myelodysplastic Syndrome;Essential Thrombocythemia;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Polycythemia Vera;Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase;Primary Myelofibrosis;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring SideroblastsDrug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Completed50 Years75 YearsAll77Phase 2United States;Denmark;Germany;Italy
6NCT00587054
(ClinicalTrials.gov)
June 200121/12/2007Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic DisordersPhase II Trial of Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Treatment of Adult Patients (>18 Years) With Lymphohematopoietic DisordersAllogeneic Stem Cell Transplant;Leukemia;Non-Hodgkins;Lymphoblastic Lymphoma;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria (PNH)Drug: cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplantMemorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI)Completed18 Years55 YearsAll129Phase 2United States

65. 原発性免疫不全症候群


臨床試験数 : 500 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04965597
(ClinicalTrials.gov)
April 19, 202221/6/2021Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure DiseasesBone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond SyndromeDrug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life AssessmentFred Hutchinson Cancer CenterBlood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)Recruiting1 Year49 YearsAll40Phase 2United States
2NCT03597594
(ClinicalTrials.gov)
September 8, 202119/6/2018Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)Severe Combined ImmunodeficiencyDrug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte InfusionSt. Jude Children's Research HospitalNULLRecruiting2 MonthsN/AAll42Phase 1/Phase 2United States
3NCT04339777
(ClinicalTrials.gov)
September 22, 20208/4/2020Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of ImmunityA Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of ImmunityLymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency DisordersDrug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irradiation;Procedure: Allogeneic HSCT;Drug: Tacrolimus (Tacro);Drug: Mycophenolate mofetil (MMF);Drug: Cyclophosphamide (Cytoxan)National Cancer Institute (NCI)NULLRecruiting4 Years69 YearsAll66Phase 2United States
4EUCTR2018-003842-18-IT
(EUCTR)
08/01/201919/11/2018Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS)A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. Wiskott-Aldrich Syndrome
MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: OTL-103 Dispersion for Infusion
Product Code: OTL-103
INN or Proposed INN: Other hematological Agents
Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
Other descriptive name: NA
Trade Name: Fludarabina Accord
INN or Proposed INN: FLUDARABINE
Other descriptive name: NA
Trade Name: MabThera
INN or Proposed INN: RITUXIMAB
Other descriptive name: NA
Trade Name: Mozobil,
INN or Proposed INN: plerixafor
Other descriptive name: PLERIXAFOR
Trade Name: MYELOSTIM
Product Name: granulocyte colony stimulating factor (G-CSF)
INN or Proposed INN:
Orchard Therapeutics Ltd.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
6Phase 3Italy
5EUCTR2018-001489-41-SE
(EUCTR)
02/07/201808/05/2018A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis congenita / telomere disease, inherited genetic conditions.Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease Dyskeratosis congenita / telomere disease
MedDRA version: 20.0;Level: PT;Classification code 10001756;Term: Allogenic bone marrow transplantation therapy;System Organ Class: 10042613 - Surgical and medical procedures ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Lemtrada
INN or Proposed INN: Alemtuzumab
Other descriptive name: ALEMTUZUMAB
Trade Name: Fludarabine
INN or Proposed INN: FLUDARABINE
Karolinska UniversitetssjukhusetNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
40 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Sweden
6NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchActive, not recruiting3 Months39 YearsAll6Phase 1/Phase 2United States
7JPRN-UMIN000030806
2018/01/1715/01/2018A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich SyndromeA phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - Hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome Wiskott-Aldrich syndromeWASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan.
1. Rituximab (day-22)
375 mg/m2

2. Preconditioning chemotherapy
Fludarabine 30mg/m2 x 2 (day-3, day-2)
Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours)

3. Infusion of WASP cDNA-transduced CD34 positive HSC
5 x 10^6/kg (at least 3 x 10^6/kg)
National Center for Child Heath and DevelopmentNULLComplete: follow-up continuingNot applicableNot applicableMale3Phase 1,2Japan
8NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNULLActive, not recruiting1 Year75 YearsAll31Phase 2United States
9JPRN-UMIN000019532
2015/12/0101/12/2015Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseasesClinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases - HSCT for ataxia telangiectasia and related diseases ataxia telangiectasiaDNA damage response disorderfludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4Graduate Schoool,Dept. of Community Pediatrics,Perinatal and Maternal Medicine,Tokyo Medical and Dental UniversityNULLRecruitingNot applicableNot applicableMale and Female5Phase 2Japan
10NCT02162420
(ClinicalTrials.gov)
January 10, 201510/6/2014Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaHematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLRecruiting0 Years70 YearsAll50N/AUnited States
11NCT02282904
(ClinicalTrials.gov)
October 23, 20144/11/2014Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant CyclophosphamideHaploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant CyclophosphamideChronic Granulomatous DiseaseDrug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: BusulfanNational Institute of Allergy and Infectious Diseases (NIAID)NULLTerminated2 Years65 YearsAll7Phase 1/Phase 2United States
12NCT01962415
(ClinicalTrials.gov)
February 4, 201410/10/2013Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTA Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults =55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell TransplantationPrimary Immunodeficiency (PID);Congenital Bone Marrow Failure Syndromes;Inherited Metabolic Disorders (IMD);Hereditary Anemias;Inflammatory ConditionsDrug: Hydroxyurea;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Melphalan;Drug: ThiotepaPaul SzabolcsNULLRecruiting2 Months55 YearsAll100Phase 2United States
13NCT01821781
(ClinicalTrials.gov)
March 201319/3/2013Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineNULLRecruitingN/A21 YearsAll20Phase 2United States
14NCT01652092
(ClinicalTrials.gov)
September 4, 201225/7/2012Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesAllogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesSCID;Omenn's Syndrome;Reticular Dysgenesis;Wiskott-Aldrich Syndrome;Bare Lymphocyte Syndrome;Common Variable Immunodeficiency;Chronic Granulomatous Disease;CD40 Ligand Deficiency;Hyper IgM Syndrome;X-linked Lymphoproliferative Disease;Hemophagocytic Lymphohistiocytosis;Griscelli Syndrome;Chediak-Higashi Syndrome;Langerhan's Cell HistiocytosisDrug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNAMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A50 YearsAll30N/AUnited States
15NCT01659606
(ClinicalTrials.gov)
July 20126/8/2012Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaRadiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere DiseaseDyskeratosis Congenita;Hoyeraal Hreidarsson Syndrome;Revesz Syndrome;Aplastic AnemiaBiological: alemtuzumab;Drug: Fludarabine;Drug: Cyclosporins;Drug: Mycophenolate mofetil;Drug: TacrolimusBoston Children's HospitalDana-Farber Cancer Institute;Children's Hospital Medical Center, Cincinnati;Children's Hospital Los Angeles;Fred Hutch/University of Washington/Seattle Children's Cancer Consortium;Baylor College of Medicine;Children's Hospital of Philadelphia;University of Wisconsin, Madison;Karolinska University Hospital;Hackensack Meridian Health;Duke University;Oslo University Hospital;Children's Mercy Hospital Kansas City;Mayo Clinic;University of Chicago;Massachusetts General HospitalActive, not recruiting30 Days65 YearsAll40Phase 2United States;Norway;Sweden
16NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;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 Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;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 Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
17NCT01338675
(ClinicalTrials.gov)
January 20114/3/2011Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)Chronic Granulomatous DiseaseDrug: BusulfanSeoul National University HospitalNULLRecruitingN/AN/ABoth5Phase 1/Phase 2Korea, Republic of
18NCT00579137
(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
19NCT00455312
(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
20NCT00885833
(ClinicalTrials.gov)
February 200721/4/2009Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich SyndromeWiskott-Aldrich SyndromeDrug: Fludarabine, Busulfan, ThymoglobulinThe Korean Society of Pediatric Hematology OncologyNULLCompleted1 Year25 YearsBoth5Phase 1/Phase 2Korea, Republic of
21NCT00358657
(ClinicalTrials.gov)
May 24, 200628/7/2006Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited DisordersHLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States
22NCT00160355
(ClinicalTrials.gov)
May 20058/9/2005Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich SyndromeHaploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot StudyWiskott-Aldrich SyndromeProcedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, ThiotepaSt. Jude Children's Research HospitalNULLCompletedN/A18 YearsMale4Phase 1United States
23NCT00295971
(ClinicalTrials.gov)
April 200523/2/2006Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency DiseaseStem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency DiseasesCongenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiationUniversity of California, San FranciscoNational Cancer Institute (NCI)Completed1 Year17 YearsBoth21Phase 1United States
24NCT00301834
(ClinicalTrials.gov)
January 20059/3/2006Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A21 YearsAll35Phase 2United States
25NCT00228852
(ClinicalTrials.gov)
April 200427/9/2005IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell ImmunodeficiencyPhase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell ImmunodeficiencyT-Cell Immune Deficiency Diseases;Severe Combined ImmunodeficiencyDrug: Busulfan, Fludarabine and ATGEmory UniversityNULLCompletedN/AN/ABothPhase 1/Phase 2United States
26NCT00578643
(ClinicalTrials.gov)
March 200419/12/2007Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous DiseaseHLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous DiseaseChronic Granulomatous DiseaseDrug: Busulfan;Biological: Alemtuzumab;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Cyclosporine;Procedure: Stem Cell InfusionBaylor College of MedicineNULLCompletedN/AN/AAll15Phase 2United States
27NCT00176852
(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
28NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityNULLRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
29NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A17 YearsBoth40Phase 1/Phase 2United States
30NCT00176878
(ClinicalTrials.gov)
June 200012/9/2005Stem Cell Transplant for Bone Marrow Failure SyndromesBone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure DisordersDiamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond SyndromeProcedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLCompletedN/A35 YearsAll10Phase 2/Phase 3United States

85. 特発性間質性肺炎


臨床試験数 : 627 薬物数 : 443 - (DrugBank : 120) / 標的遺伝子数 : 99 - 標的パスウェイ数 : 212
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03500731
(ClinicalTrials.gov)
April 19, 201830/3/2018Lung and Bone Marrow Transplantation for Lung and Bone Marrow FailureLung Transplant in Tandem With Bone Marrow Transplant for Combined Lung and Bone Marrow FailureIdiopathic Pulmonary Fibrosis;Emphysema or COPDBiological: CD3/CD19 negative hematopoietic stem cells;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Thiotepa;Drug: G-CSF;Drug: HydroxyureaPaul SzabolcsNULLRecruiting18 Years60 YearsAll8Phase 1/Phase 2United States

96. クローン病


臨床試験数 : 2,442 薬物数 : 1,278 - (DrugBank : 248) / 標的遺伝子数 : 142 - 標的パスウェイ数 : 209
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04224558
(ClinicalTrials.gov)
December 15, 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 CenterNULLRecruiting13 Years28 YearsAll15Phase 1/Phase 2United States
2NCT04154735
(ClinicalTrials.gov)
November 20198/3/2019Autologous Transplant Targeted Against Crohn'sAutologous Hematopoietic Stem Cell Transplant for Crohn's DiseaseCrohn's DiseaseDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Mesna;Drug: Alemtuzumab;Drug: G-CSF;Drug: Rifaximin;Drug: TacrolimusNorthwestern UniversityNULLWithdrawn18 Years49 YearsAll0Phase 2United States
3EUCTR2017-002545-30-GB
(EUCTR)
06/11/201707/06/2018Controlled trial to assess low intensity autologous stem cell transplant in refractory Crohn's DiseaseAutologous Stem cell Transplantation In refractory Crohn's disease - Low Intensity Therapy Evaluation - ASTIClite Refractory Crohn's Disease
MedDRA version: 20.0;Level: PT;Classification code 10011401;Term: Crohn's disease;System Organ Class: 10017947 - Gastrointestinal disorders;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Trade Name: Cyclophosphamide
Product Name: Cyclophosphamide
INN or Proposed INN: Cyclophosphamide monohydrate
INN or Proposed INN: Cyclophosphamide monohydrate
Trade Name: Filgrastim
Product Name: Filgrastim
INN or Proposed INN: Filgrastim
Trade Name: Fludarabine
Product Name: Fludarabine
INN or Proposed INN: Fludarabine phosphate
Trade Name: Thymoglobuline
Product Name: Thymoglobuline
INN or Proposed INN: Rabbit anti-human thymocyte immunoglobulin
Barts Health NHS TrustNULLNot RecruitingFemale: yes
Male: yes
99Phase 3United Kingdom
4NCT01570348
(ClinicalTrials.gov)
July 17, 20122/4/2012Crohn's Allogeneic Transplant StudyAllogeneic Hematopoietic Cell Transplantation for Patients With Treatment-Refractory Crohn's Disease: A Phase 2 StudyCrohn DiseaseProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Drug: Mycophenolic Acid;Other: Quality-of-Life Assessment;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Institutes of Health (NIH)Terminated18 Years60 YearsAll2Phase 2United States

164. 眼皮膚白皮症


臨床試験数 : 15 薬物数 : 54 - (DrugBank : 34) / 標的遺伝子数 : 35 - 標的パスウェイ数 : 141
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT01821781
(ClinicalTrials.gov)
March 201319/3/2013Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineNULLRecruitingN/A21 YearsAll20Phase 2United States
2NCT01652092
(ClinicalTrials.gov)
September 4, 201225/7/2012Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesAllogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesSCID;Omenn's Syndrome;Reticular Dysgenesis;Wiskott-Aldrich Syndrome;Bare Lymphocyte Syndrome;Common Variable Immunodeficiency;Chronic Granulomatous Disease;CD40 Ligand Deficiency;Hyper IgM Syndrome;X-linked Lymphoproliferative Disease;Hemophagocytic Lymphohistiocytosis;Griscelli Syndrome;Chediak-Higashi Syndrome;Langerhan's Cell HistiocytosisDrug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNAMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A50 YearsAll30N/AUnited States
3NCT00176865
(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

283. 後天性赤芽球癆


臨床試験数 : 19 薬物数 : 36 - (DrugBank : 23) / 標的遺伝子数 : 20 - 標的パスウェイ数 : 102
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04965597
(ClinicalTrials.gov)
April 19, 202221/6/2021Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure DiseasesBone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond SyndromeDrug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life AssessmentFred Hutchinson Cancer CenterBlood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)Recruiting1 Year49 YearsAll40Phase 2United States
2NCT03333486
(ClinicalTrials.gov)
December 7, 20172/11/2017Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerA Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem CellsAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body IrradiationRoswell Park Cancer InstituteNULLActive, not recruiting1 Year75 YearsAll31Phase 2United States

284. ダイアモンド・ブラックファン貧血


臨床試験数 : 36 薬物数 : 95 - (DrugBank : 34) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03653338
(ClinicalTrials.gov)
August 2, 20182/8/2018T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other AnemiasT-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent AnemiasSickle Cell Anemia;Beta-thalassemia Major;Diamond-blackfan AnemiaBiological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: ThiotepaPaul SzabolcsNULLRecruiting5 Years40 YearsAll5Phase 1/Phase 2United States
2NCT03513328
(ClinicalTrials.gov)
June 15, 201819/4/2018Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchActive, not recruiting3 Months39 YearsAll6Phase 1/Phase 2United States
3NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;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 Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;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 Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
4NCT00301834
(ClinicalTrials.gov)
January 20059/3/2006Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A21 YearsAll35Phase 2United States
5NCT00176852
(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
6NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A17 YearsBoth40Phase 1/Phase 2United States
7NCT00176878
(ClinicalTrials.gov)
June 200012/9/2005Stem Cell Transplant for Bone Marrow Failure SyndromesBone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure DisordersDiamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond SyndromeProcedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLCompletedN/A35 YearsAll10Phase 2/Phase 3United States

285. ファンコニ貧血


臨床試験数 : 62 薬物数 : 93 - (DrugBank : 30) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 144
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04784052
(ClinicalTrials.gov)
December 7, 20212/3/2021Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing JSP191 AntibodyTCRaß+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi AnemiaFanconi AnemiaDrug: JSP191;Device: CliniMACS Prodigy System;Biological: Depleted Stem Cell Transplant;Biological: Rabbit Anti-Thymoglobulin (rATG);Drug: Cyclophosphamide;Drug: Fludarabine;Drug: RituximabRajni AgarwalNULLRecruiting2 YearsN/AAll12Phase 1/Phase 2United States
2NCT03579875
(ClinicalTrials.gov)
November 13, 201825/5/2018T Cell Receptor a/ß TCD HCT in Patients With Fanconi AnemiaT Cell Receptor Alpha/Beta T Cell Depleted (a/ß TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA)Fanconi Anemia;Severe Aplastic Anemia;Myelodysplastic SyndromesDrug: Total Body Irradiation (TBI) (Plan 1);Drug: Cyclophosphamide (CY) (Plan 1);Drug: Fludarabine (FLU);Drug: Methylprednisolone (MP);Device: Donor mobilized PBSC infusion;Drug: G-CSF;Drug: Cyclophosphamide (CY) (Plan 2);Drug: Rituximab;Drug: BusulfanMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A65 YearsAll48Phase 2United States
3NCT03600909
(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 ConsortiumCompleted1 MonthN/AAll3Phase 2United States
4NCT02143830
(ClinicalTrials.gov)
April 201428/4/2014HSCT for Patients With Fanconi Anemia Using Risk-Adjusted ChemotherapyA Phase II Trial of HSCT 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;Severe Marrow Failure;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)Drug: Busulfan;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: rabbit ATG;Drug: G-CSF;Biological: Peripheral blood stem cellChildren's Hospital Medical Center, CincinnatiFred Hutchinson Cancer Center;Memorial Sloan Kettering Cancer CenterRecruiting3 MonthsN/AAll70Phase 2United States
5NCT02127905
(ClinicalTrials.gov)
March 201119/10/2012Unrelated HSCT in Patients With Fanconi AnemiaA Study of Total Body Irradiation, Cyclophosphamide and Fludarabine Followed by Alternated Donor Hematopoietic Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaBiological: CD34+ selected cellsNeena Kapoor, M.D.NULLWithdrawn8 Weeks21 YearsAll0N/AUnited States
6NCT01316549
(ClinicalTrials.gov)
January 1, 201114/3/2011Study of Fludarabine Drug Exposure in Pediatric Bone Marrow TransplantationPopulation Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell TransplantationHematologic Malignancies;Nonmalignant Diseases;Immunodeficiencies;Hemoglobinopathies;Genetic Inborn Errors of Metabolism;Fanconi Anemia;Thalassemia;Sickle Cell DiseaseDrug: FludarabineUniversity of California, San FranciscoThrasher Research FundCompletedN/A17 YearsAll67United States
7NCT01082133
(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
8NCT00987480
(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
9NCT01071239
(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
10NCT00856388
(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
11NCT00453388
(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
12NCT00352976
(ClinicalTrials.gov)
May 18, 200614/7/2006TBI Dose De-escalation for Fanconi AnemiaTotal Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell TransplantationFanconi AnemiaDrug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Bone Marrow Transplantation;Drug: Mycophenolate Mofetil;Drug: SirolimusMasonic Cancer Center, University of MinnesotaNULLCompletedN/AN/AAll88Phase 2/Phase 3United States
13NCT00084695
(ClinicalTrials.gov)
September 200310/6/2004Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant DiseasesThe Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem CellsChildhood Langerhans Cell Histiocytosis;Fanconi Anemia;Leukemia;Lymphoma;Myelodysplastic Syndromes;Neuroblastoma;Sarcoma;Unspecified Childhood Solid Tumor, Protocol SpecificBiological: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: melphalan;Drug: methylprednisolone;Radiation: radiation therapyMilton S. Hershey Medical CenterNULLRecruitingN/A21 YearsBoth25Phase 2United States
14NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityNULLRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
15NCT00258427
(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
16NCT00586274
(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
17NCT00053989
(ClinicalTrials.gov)
January 29, 20025/2/2003NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/DisordersNon-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and DisordersChronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic AnemiaBiological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Biological: sargramostim;Biological: therapeutic allogeneic lymphocytes;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationRoswell Park Cancer InstituteNULLCompleted4 Years75 YearsAll41Phase 2United States
18NCT00590460
(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
19NCT00595127
(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
20NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A17 YearsBoth40Phase 1/Phase 2United States
21NCT00630253
(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
22NCT00093743
(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

286. 遺伝性鉄芽球性貧血


臨床試験数 : 7 薬物数 : 20 - (DrugBank : 10) / 標的遺伝子数 : 8 - 標的パスウェイ数 : 43
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04965597
(ClinicalTrials.gov)
April 19, 202221/6/2021Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure DiseasesBone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond SyndromeDrug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life AssessmentFred Hutchinson Cancer CenterBlood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)Recruiting1 Year49 YearsAll40Phase 2United States

326. 大理石骨病


臨床試験数 : 18 薬物数 : 43 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 82
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1NCT02171104
(ClinicalTrials.gov)
July 10, 201420/6/2014MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic DisordersBiological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D)Masonic Cancer Center, University of MinnesotaNULLRecruitingN/A55 YearsAll100Phase 2United States
2NCT01087398
(ClinicalTrials.gov)
September 200913/3/2010Hematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisHematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisOsteopetrosisDrug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)Tehran University of Medical SciencesNULLRecruitingN/A5 YearsBoth10Phase 2/Phase 3Iran, Islamic Republic of
3NCT00775931
(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
4NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityNULLRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States