- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- Ravulizumab, Pozelimab, Cemdisiran, Eculizumab
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 202 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Whittier, California
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Showing 25–48
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- 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
- Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Cyclophosphamide, Peripheral Blood Stem Cells
- Drug · Other
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 4 Years to 75 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Mantle Cell Lymphoma, Multiple Myeloma, Waldenstrom Macroglobulinemia, Breast Cancer, Renal Cell Carcinoma, Melanoma, Sarcoma, Ovarian Cancer, Thymoma
- Interventions
- Nonmyeloablative Allogeneic Transplant
- Procedure
- Lead sponsor
- Scripps Health
- Other
- Eligibility
- 18 Years to 72 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2015
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Complement Mediated Diseases
- Interventions
- AMY-101
- Drug
- Lead sponsor
- Amyndas Pharmaceuticals S.A.
- Industry
- Eligibility
- 18 Years to 60 Years · Male only
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017
- U.S. locations
- 1
- States / cities
- High Point, North Carolina
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis (gMG), Neuromyelitis Optica Spectrum Disorder (NMOSD)
- Interventions
- Ultomiris
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Female only
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2034
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
- Interventions
- Pegcetacoplan
- Drug
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Interventions
- CliniMACs
- Device
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Up to 25 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2029
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- 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
- 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
- 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
- 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
- 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
- Interventions
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
- Drug · Other · Procedure + 1 more
- Lead sponsor
- Roswell Park Cancer Institute
- Other
- Eligibility
- 1 Year to 75 Years
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- Buffalo, New York
- Conditions
- Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria, Hodgkin's Lymphoma, Non-Hodgkin Lymphoma
- Interventions
- Systematic chemotherapy and antibodies, Allogeneic stem cell transplantation, Miltenyi CliniMACS
- Drug · Procedure · Device
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- 2 Years to 21 Years
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2009
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH
- Interventions
- Not listed
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 3
- States / cities
- Englewood, Colorado • Weston, Florida • Scarborough, Maine
- Conditions
- Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia (JMML), Hemoglobinuria, Paroxysmal Nocturnal (PNH), Hodgkin Lymphoma, Lymphoma, Non-Hodgkin (NHL), Myelodysplastic Syndrome (MDS)
- Interventions
- CliniMACS, Stem cell transplantation, Fludarabine, Thioplex®, L-phenylalanine mustard, Mycophenolate mofetil, Rituxan™, Alemtuzumab, Cyclophosphamide, Anti-thymocyte globulin (Rabbit), G-CSF, Muromonab
- Device · Procedure · Drug
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 73 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2020
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Interventions
- ABP 959, Eculizumab
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- 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
- 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
- Preeclampsia, Severe Preeclampsia, Eculizumab, HELLP, HELLP Syndrome, HELLP Syndrome Second Trimester, Pregnancy Related, AHUS, PNH, Complement Regulatory Factor Defect, Complement Abnormality, HELLP Syndrome Third Trimester
- Interventions
- Eculizumab
- Drug
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 13 Years and older · Female only
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Pegcetacoplan
- Drug
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 7
- States / cities
- Los Angeles, California • Miami Lakes, Florida • Louisville, Kentucky + 4 more
- 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
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- LNP023, Eculizumab, Ravulizumab
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 97 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 5
- States / cities
- Duarte, California • Orange, California • Augusta, Georgia + 2 more
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- ALXN1102, ALXN1103
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Los Angeles, California