62. Paroxysmal nocturnal hemoglobinuria Clinical trials / Disease details
Clinical trials : 292 / Drugs : 151 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 108
| 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 | NCT04965597 (ClinicalTrials.gov) | April 19, 2022 | 21/6/2021 | Treosulfan-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 Diseases | Bone 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 Syndrome | Drug: 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 Assessment | Fred Hutchinson Cancer Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 2 | NCT03333486 (ClinicalTrials.gov) | December 7, 2017 | 2/11/2017 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated 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 Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | NULL | Active, not recruiting | 1 Year | 75 Years | All | 31 | Phase 2 | United States |
| 3 | NCT01529827 (ClinicalTrials.gov) | February 28, 2012 | 6/2/2012 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Accelerated 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 Syndrome | Drug: 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 transplantation | Roswell Park Cancer Institute | NULL | Completed | 3 Years | 75 Years | All | 94 | Phase 2 | United States |
| 4 | NCT00856388 (ClinicalTrials.gov) | January 14, 2009 | 4/3/2009 | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders | A Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body Irradiation | Accelerated 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 Macroglobulinemia | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulin | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Completed | 3 Years | 75 Years | All | 62 | N/A | United States |
| 5 | NCT00397813 (ClinicalTrials.gov) | January 2006 | 9/11/2006 | Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders | Low-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 Trial | Atypical 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 Sideroblasts | Drug: 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 Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Completed | 50 Years | 75 Years | All | 77 | Phase 2 | United States;Denmark;Germany;Italy |