Thiotepa    (DrugBank: Thiotepa)

11 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

13. 多発性硬化症/視神経脊髄炎 [臨床試験数:3,050,薬物数:2,147(DrugBank:348),標的遺伝子数:244,標的パスウェイ数:228
Searched query = "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 3,050 trial found
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
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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
Italy

19. ライソゾーム病 [臨床試験数:784,薬物数:673(DrugBank:101),標的遺伝子数:68,標的パスウェイ数:184
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 784 trial found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
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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 ResearchRecruiting3 Months39 YearsAll40Phase 1/Phase 2United States

20. 副腎白質ジストロフィー [臨床試験数:49,薬物数:86(DrugBank:29),標的遺伝子数:18,標的パスウェイ数:112
Searched query = "Adrenoleukodystrophy", "Adrenomyeloneuropathy", "AMN"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 49 trial found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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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 ResearchRecruiting3 Months39 YearsAll40Phase 1/Phase 2United States

28. 全身性アミロイドーシス [臨床試験数:212,薬物数:234(DrugBank:72),標的遺伝子数:54,標的パスウェイ数:170
Searched query = "Systemic amyloidosis", "AL amyloidosis", "Immunoglobulin light chain amyloidosis", "Amyloid light-chain amyloidosis", "Familial amyloidosis", "Familial amyloid polyneuropathy", "Senile transthyretin amyloidosis", "Senile TTR amyloidosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 212 trial found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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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:466,薬物数:536(DrugBank:142),標的遺伝子数:110,標的パスウェイ数:210
Searched query = "Scleroderma", "Systemic sclerosis", "SSc", "dcSSc", "lcSSc"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 466 trial found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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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 IrradiationPaul SzabolcsNULLRecruiting16 Years70 YearsAll8Phase 2United States

60. 再生不良性貧血 [臨床試験数:218,薬物数:362(DrugBank:81),標的遺伝子数:39,標的パスウェイ数:155
Searched query = "Aplastic anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
3 / 218 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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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:413,薬物数:581(DrugBank:97),標的遺伝子数:68,標的パスウェイ数:202
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
6 / 413 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT03597594
(ClinicalTrials.gov)
December 202019/6/2018Haplocompatible 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 ResearchRecruiting3 Months39 YearsAll40Phase 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
No.TrialIDDate_
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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:514,薬物数:377(DrugBank:108),標的遺伝子数:97,標的パスウェイ数:204
Searched query = "Idiopathic interstitial pneumonia", "IIPs", "Idiopathic pulmonary fibrosis", "IPF", "Usual interstitial pneumonia", "UIP", "Non-specific interstitial pneumonia", "NSIP", "Acute interstitial pneumonia", "AIP", "Diffuse alveolar damage", "DAD", "COP", "Organizing pneumonia", "OP", "Desquamative interstitial pneumonia", "DIP", "Respiratory bronchiolitis - associated interstitial lung disease", "RB-ILD", "Lymphocytic interstitial pneumonia", "LIP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 514 trial found
No.TrialIDDate_
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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:2,209,薬物数:1,276(DrugBank:240),標的遺伝子数:166,標的パスウェイ数:210
Searched query = "Crohn disease", "Terminal ileitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 2,209 trial found
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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:57(DrugBank:34),標的遺伝子数:34,標的パスウェイ数:136
Searched query = "Oculocutaneous albinism", "Hermansky-Pudlak syndrome", "Chediak-Higashi syndrome", "Griscelli syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 15 trial found
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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:37,薬物数:110(DrugBank:34),標的遺伝子数:22,標的パスウェイ数:121
Searched query = "Diamond-Blackfan anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 37 trials found
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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 ResearchRecruiting3 Months39 YearsAll40Phase 1/Phase 2United States