284. ダイアモンド・ブラックファン貧血 Diamond-Blackfan anemia Clinical trials / Disease details
臨床試験数 : 37 / 薬物数 : 110 - (DrugBank : 34) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 124
Showing 1 to 10 of 37 diseases
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT04269889 (ClinicalTrials.gov) | December 1, 2020 | 13/2/2020 | Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag | Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag | Anemia, Diamond-Blackfan | Drug: Eltrombopag | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Recruiting | 2 Years | N/A | All | 30 | Phase 1/Phase 2 | United States |
2 | NCT04099966 (ClinicalTrials.gov) | April 1, 2020 | 18/9/2019 | AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B ... | Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588 Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing ... | Acute Leukemia;Severe Aplastic Anemia;Non-hodgkin Lymphoma;Hodgkin Lymphoma;Kostmann;Diamond Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Sickle Cell Disease;Beta-Thalassemia Acute Leukemia;Severe Aplastic Anemia;Non-hodgkin Lymphoma;Hodgkin Lymphoma;Kostmann;Diamond Blackfa ... | Drug: alpha beta depletion | Mitchell Cairo | NULL | Not yet recruiting | N/A | 30 Years | All | 20 | Phase 2 | United States |
3 | NCT03966053 (ClinicalTrials.gov) | September 13, 2019 | 23/5/2019 | The Use of Trifluoperazine in Transfusion Dependent DBA | Phase I/II, Open Label Study to Determine Safety of Trifluoperazine (TFP) in Adults With Red Blood Cell Transfusion-Dependent Diamond Blackfan Anemia Phase I/II, Open Label Study to Determine Safety of Trifluoperazine (TFP) in Adults With Red Blood C ... | Diamond Blackfan Anemia;Pure Red Cell Aplasia | Drug: Trifluoperazine | Adrianna Vlachos | NULL | Terminated | 18 Years | 65 Years | All | 2 | Phase 1/Phase 2 | United States |
4 | NCT03653338 (ClinicalTrials.gov) | August 2, 2018 | 2/8/2018 | T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Ane ... | T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cel ... | Sickle Cell Anemia;Beta-thalassemia Major;Diamond-blackfan Anemia | Biological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Thiotepa Biological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Dr ... | Paul Szabolcs | NULL | Recruiting | 5 Years | 40 Years | All | 5 | Phase 1/Phase 2 | United States |
5 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for ... | Bone 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;Adrenoleukodystrophy Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutro ... | Drug: Thiotepa--single daily dose;Drug: Thiotepa--escalated dose | University of Florida | Live Like Bella Pediatric Cancer Research | Recruiting | 3 Months | 39 Years | All | 40 | Phase 1/Phase 2 | United States |
6 | NCT03733249 (ClinicalTrials.gov) | January 2017 | 2/11/2018 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Don ... | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplas ... | Drug: Rimiducid;Biological: rivogenlecleucel | Bellicum Pharmaceuticals | NULL | Enrolling by invitation | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;Saudi Arabia;United Kingdom |
7 | NCT02179359 (ClinicalTrials.gov) | September 2, 2014 | 27/6/2014 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopat ... | Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic Disorders Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxy ... | Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative Regimen Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablat ... | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 25 | N/A | United States |
8 | NCT02386267 (ClinicalTrials.gov) | September 2014 | 18/2/2015 | L-leucine in Diamond Blackfan Anemia Patients | Therapeutic Use of the Amino Acid Leucine in the Treatment of Transfusion-Dependent Diamond Blackfan Anemia Patients Therapeutic Use of the Amino Acid Leucine in the Treatment of Transfusion-Dependent Diamond Blackfan ... | Diamond Blackfan Anemia | Drug: L-leucine | Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmit ... | NULL | Recruiting | 1 Year | 20 Years | Both | 30 | Phase 2 | Russian Federation |
9 | NCT02065869 (ClinicalTrials.gov) | April 2014 | 13/2/2014 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor A ... | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplas ... | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 18 Years | All | 193 | Phase 2 | Italy;United Kingdom;Germany;Spain;United States |
10 | NCT01917708 (ClinicalTrials.gov) | January 2014 | 24/7/2013 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic He ... | Hurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell Disease Hurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomato ... | Drug: Abatacept | Emory University | NULL | Completed | N/A | 21 Years | All | 10 | Phase 1 | United States |