- Conditions
- Hemoglobinuria, Paroxysmal
- Interventions
- eculizumab
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 1
- States / cities
- Livingston, New Jersey
Search Results
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Showing 1–24
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- 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
- Interventions
- Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil, Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
- Drug · Other · Procedure + 1 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- 50 Years to 75 Years
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2018
- U.S. locations
- 3
- States / cities
- Salt Lake City, Utah • Seattle, Washington
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Zilucoplan (RA101495)
- Drug
- Lead sponsor
- Ra Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Dallas, Texas
- Conditions
- Bone Marrow Failure Disorders, VEXAS Syndrome, Hemoglobinurea, Paroxysmal, Myelodysplastic Syndromes, Bone Marrow Diseases
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 2 Years to 120 Years
- Enrollment
- 1,400 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
- Interventions
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Device · Other
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 4 Years to 80 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2028
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland • Bethesda, Maryland
- Conditions
- Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant
- Drug
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 129 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2011
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Ravulizumab
- Biological
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Up to 17 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia • Milwaukee, Wisconsin
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Interventions
- Danicopan, Soliris/Ultomiris
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Secondary AML, Myelodysplastic Syndrome (MDS), Secondary MDS, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia (JMML), Paroxysmal Nocturnal Hemoglobinuria (PNH), Lymphoma, Non-Hodgkin, Hodgkin Disease
- Interventions
- Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies
- Procedure · Device · Drug
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- 2 Years to 21 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2009
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- 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;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;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
- Interventions
- fludarabine phosphate, melphalan, total-body irradiation, allogeneic hematopoietic stem cell transplantation, anti-thymocyte globulin
- Drug · Radiation · Procedure + 1 more
- Lead sponsor
- Roswell Park Cancer Institute
- Other
- Eligibility
- 3 Years to 75 Years
- Enrollment
- 62 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2019
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Hemoglobinuria, Paroxysmal
- Interventions
- Not listed
- Lead sponsor
- University of Utah
- Other
- Eligibility
- 7 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2010
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Paroxysmal Hemoglobinuria
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2005
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Iptacopan
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2024
- U.S. locations
- 9
- States / cities
- Duarte, California • Los Angeles, California • Miami Lakes, Florida + 6 more
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Cemdisiran, Eculizumab, Pozelimab, Ravulizumab
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Whittier, California
- Conditions
- Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal, Red-Cell Aplasia, Pure, Purpura, Thrombocytopenic, Leukemia, Lymphocytic
- Interventions
- Not listed
- Lead sponsor
- Office of Rare Diseases (ORD)
- NIH
- Eligibility
- 11 Years and older
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Tampa, Florida • Cleveland, Ohio + 1 more
- Conditions
- PNH
- Interventions
- Pegcetacoplan
- Drug
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 137 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Denver, Colorado • Jacksonville, Florida + 6 more
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Shwachman-Diamond Syndrome, Hematologic Neoplasm With Germline GATA2 Mutation, Hematologic Neoplasm With Germline SAMD9 Mutation, Hematologic Neoplasm With Germline SAMD9L Mutation
- Interventions
- Treosulfan, Fludarabine Phosphate, Tacrolimus, Methotrexate, Lapine T-Lymphocyte Immune Globulin, Peripheral Blood Stem Cell Transplantation, Allogeneic Bone Marrow Transplantation, Quality-of-Life Assessment, Echocardiography, Multigated Acquisition Scan, Bone Marrow Biopsy, Bone Marrow Aspiration, Biospecimen Collection, X-Ray Imaging, Computed Tomography
- Drug · Biological · Procedure + 1 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- 1 Year to 49 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 24
- States / cities
- Los Angeles, California • San Diego, California • San Francisco, California + 20 more
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, 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;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, Paroxysmal Nocturnal Hemoglobinuria, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia, Refractory Anemia With Ringed Sideroblasts, Relapsing Chronic Myelogenous Leukemia, Thrombocytopenia, Untreated Adult Acute Myeloid Leukemia
- Interventions
- bryostatin 1, sargramostim, laboratory biomarker analysis, pharmacological study
- Drug · Biological · Other
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2001
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Zilucoplan (RA101495)
- Drug
- Lead sponsor
- Ra Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2018
- U.S. locations
- 6
- States / cities
- Duarte, California • Los Angeles, California • Chicago, Illinois + 3 more
- Conditions
- Hemoglobinuria, Paroxysmal
- Interventions
- Eculizumab
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 3
- States / cities
- Orange, California • Pensacola, Florida • Memphis, Tennessee
- Conditions
- Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, Myeloproliferative Syndromes, Chronic Myelogenous Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Multiple Myeloma, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Large Granulocytic Leukemia
- Interventions
- Allogeneic transplantation
- Procedure
- Lead sponsor
- Scripps Health
- Other
- Eligibility
- 18 Years to 69 Years
- Enrollment
- 64 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2015
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Ravulizumab, Eculizumab
- Biological
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 272 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2023
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Whittier, California • Fort Worth, Texas
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 6,061 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2025
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Pegcetacoplan, Soliris
- Drug
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 19
- States / cities
- Los Angeles, California • Santa Monica, California • Denver, Colorado + 16 more