THIOTEPA ( DrugBank: Thiotepa )


12 diseases
告示番号疾患名(ページ内リンク)臨床試験数
13多発性硬化症/視神経脊髄炎1
19ライソゾーム病1
20副腎白質ジストロフィー1
28全身性アミロイドーシス1
51全身性強皮症1
60再生不良性貧血3
65原発性免疫不全症候群6
85特発性間質性肺炎1
96クローン病1
164眼皮膚白皮症1
284ダイアモンド・ブラックファン貧血2
285ファンコニ貧血1

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
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agemax
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size
PhaseCountries
1EUCTR2007-000586-38-IT
(EUCTR)
21/12/200710/01/2008HIGH-DOSE IMMUNOSUPPRESSION WITH THIOTEPA AND CYCLOPHOSPHAMIDE FOLLOWED BY AUTOLOGOUS STEM CELL TRANSPLANTATION IN MULTIPLE SCLEROSIS - NDHIGH-DOSE IMMUNOSUPPRESSION WITH THIOTEPA AND CYCLOPHOSPHAMIDE FOLLOWED BY AUTOLOGOUS STEM CELL TRANSPLANTATION IN MULTIPLE SCLEROSIS - ND PATIENTS AFFECTED BY MULTIPLE SCLEROSIS
MedDRA version: 6.1;Level: HLT;Classification code 10052785
Trade Name: THIOPLEX
INN or Proposed INN: Thiotepa
Trade Name: ENDOXAN BAXTER
INN or Proposed INN: Cyclophosphamide
Trade Name: NEUPOGEN
INN or Proposed INN: Filgrastim
OSPEDALE S. RAFFAELENULLNot RecruitingFemale: yes
Male: yes
30Phase 2Italy

19. ライソゾーム病


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03513328
(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

20. 副腎白質ジストロフィー


臨床試験数 : 61 薬物数 : 90 - (DrugBank : 31) / 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
1NCT03513328
(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

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
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agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT00536601
(ClinicalTrials.gov)
June 29, 200627/9/2007High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid TumorsAutologous Blood and Marrow Transplantation for Hematologic Malignancies and Selected Solid TumorsAdult 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;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 Nasal Type Extranodal NK/T-cell Lymphoma;Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor (PNET);Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Intraocular Lymphoma;Nodal Marginal Zone B-cell Lymphoma;Peripheral T-cell Lymphoma;Plasma Cell Neoplasm;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 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 Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Malignant Testicular Germ Cell Tumor;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Neuroblastoma;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Regional Neuroblastoma;Splenic Marginal Zone Lymphoma;Testicular Lymphoma;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol Specific;Waldenström MacroglobulinemiaDrug: etoposide;Drug: cyclophosphamide;Drug: carmustine;Drug: melphalan;Drug: busulfan;Drug: carboplatin;Drug: thiotepa;Radiation: total-body irradiation;Procedure: autologous hematopoietic stem cell transplantation;Procedure: autologous-autologous tandem hematopoietic stem cell transplantationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Completed4 YearsN/AAll174N/AUnited 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
1NCT03630211
(ClinicalTrials.gov)
July 31, 20187/8/2018Autologous Stem Cell Transplantation in Patients With Systemic SclerosisAutologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Patients With Treatment Resistant Systemic Sclerosis (SSc)Systemic Sclerosis;Diffuse Sclerosis Systemic;Interstitial Lung Disease;Pulmonary HypertensionDrug: Cyclophosphamide;Drug: Mesna;Drug: Rituximab;Drug: Alemtuzumab;Drug: Thiotepa;Drug: GM-CSF;Drug: Intravenous immunoglobulin;Radiation: Total Body Irradiation;Drug: Anti Thymocyte GlobulinPaul SzabolcsNULLRecruiting8 Years60 YearsAll8Phase 2United 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
1EUCTR2007-001657-26-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
2NCT00003816
(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
3NCT00002718
(ClinicalTrials.gov)
November 19951/11/1999T-cell Depleted Bone Marrow and G-CSF Stimulated Peripheral Stem Cell Transplantation From Related Donors in Treating Patients With Leukemia, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, or Aplastic AnemiaA Phase II Trial of T-Cell Depleted Marrow Grafts Combined With Infusions of G-CSF Stimulated, CD34 Ceprate Stem Cell Column Selected, E-Rosette Depleted Peripheral Blood Progenitor Cells Derived From HLA Haplotype Matched Related Donors for Patients With Leukemia Lacking an HLA-Matched Related or Unrelated DonorLeukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative NeoplasmsBiological: anti-thymocyte globulin;Biological: filgrastim;Drug: cyclophosphamide;Drug: cytarabine;Drug: methylprednisolone;Drug: thiotepa;Procedure: in vitro-treated bone marrow transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: radiation therapyMemorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI)CompletedN/A49 YearsBoth31Phase 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
1NCT03597594
(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
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
3NCT01962415
(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
4NCT01821781
(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
5NCT00160355
(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
6NCT00295971
(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

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
1NCT00692939
(ClinicalTrials.gov)
June 26, 20123/6/2008Autologous Stem Cell Transplantation for Crohn's DiseaseAutologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Adult Patients With Severe Crohn's DiseaseCrohn's DiseaseBiological: autologous CD34-selected peripheral blood stem cells transplant;Drug: Alemtuzumab;Drug: ATG;Drug: Melphalan;Drug: Thiotepa;Drug: Rituximab;Drug: Cyclophosphamide;Drug: G-CSF;Drug: MesnaPaul SzabolcsNULLRecruiting10 Years60 YearsAll20Phase 1/Phase 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

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

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
1NCT03609840
(ClinicalTrials.gov)
May 24, 20175/1/2018Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant PatientsPopulation Pharmacokinetics and Pharmacodynamics of Thiotepa and TEPA in Pediatric Patients Undergoing Hematopoietic Cell Transplantation (HCT).Hematologic Malignancies;Nonmalignant Diseases;Immune Deficiency;Hemoglobinopathies;Genetic Inborn Errors of Metabolism;Fanconi Anemia;Thalassemia;Sickle Cell DiseaseDrug: ThiotepaUniversity of California, San FranciscoNULLActive, not recruitingN/A17 YearsAll25United States