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 : 36 / 薬物数 : 95 - (DrugBank : 34) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

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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
1	NCT04099966 (ClinicalTrials.gov)	April 1, 2021	18/9/2019	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 ...	Acute Leukemia;Severe Aplastic Anemia;Non-hodgkin Lymphoma;Hodgkin Lymphoma;Kostmann;Diamond Blackfa ...	Drug: alpha beta depletion	Mitchell Cairo	NULL	Recruiting	1 Day	30 Years	All	20	Phase 2	United States
2	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	Active, not recruiting	2 Years	N/A	All	15	Phase 1/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 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 Ane ...	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;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 ...	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	Active, not recruiting	3 Months	39 Years	All	6	Phase 1/Phase 2	United States
6	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 Hemoglobinopat ...	Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxy ...	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
7	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 ...	Diamond Blackfan Anemia	Drug: L-leucine	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
8	NCT02065869 (ClinicalTrials.gov)	April 2014	13/2/2014	Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell Transplant	Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Fami ...	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	187	Phase 1/Phase 2	Italy;United Kingdom;Germany;Spain;United States
9	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 He ...	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
10	NCT01362595 (ClinicalTrials.gov)	June 2013	20/5/2011	Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Dia ...	The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance Using ...	Diamond Blackfan Anemia;Blackfan Diamond Syndrome;DBA;Congenital Hypoplastic Anemia;Pure Red Cell Ap ...	Drug: leucine	Northwell Health	NULL	Active, not recruiting	2 Years	N/A	All	50	Phase 1/Phase 2	United States

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