Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 30, 2026, 11:31 AM EDT

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Showing 1–24 of 71 matching trials from the live ClinicalTrials.gov search.
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Humanized anti-Factor Bb monoclonal antibody, Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 45 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
2
States / cities
Glendale, California • Baltimore, Maryland
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
LNP023
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
4
States / cities
Atlanta, Georgia • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more
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
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
Interventions
Ravulizumab OBDS, Ravulizumab
Combination Product · Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
139 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Crovalimab, Eculizumab
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years and older
Enrollment
190 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Charlotte, North Carolina • Columbus, Ohio
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
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
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
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
Hematologic Neoplasms, Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Multiple Myeloma
Interventions
haploidentical allogeneic PBSC transp
Other
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 75 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
1
States / cities
Tucson, Arizona
Conditions
Acute Lymphoblastic Leukemias, Acute Myelocytic Leukemia, Chronic Myeloid Leukemia, Juvenile Myelomonocytic Leukemia, Myelodysplastic Syndrome, Hemoglobinuria, Paroxysmal, Non-Hodgkin Lymphoma
Interventions
Allogeneic Stem Cell Transplantation, Chemotherapy and antibodies
Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
2 Years to 21 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2009
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
Leukemia
Interventions
eculizumab
Biological
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Los Angeles, California
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
Conditions
Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
Interventions
Campath, Chemo and/or TBI Allo SCT
Drug
Lead sponsor
David Rizzieri, MD
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
2
States / cities
Orlando, Florida • Durham, 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
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
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
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;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;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
Interventions
fludarabine phosphate, melphalan, total-body irradiation, tacrolimus, mycophenolate mofetil, methotrexate, laboratory biomarker analysis, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation
Drug · Radiation · Other + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
3 Years to 75 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
1
States / cities
Buffalo, New York
Conditions
Rheologic Disease, Sickle Cell Disease, Paroxysmal Nocturnal Hemoglobinuria
Interventions
Imaging
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
19 Years and older
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Portland, Oregon
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
Danicopan, Placebo, C5 Inhibitor
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
7
States / cities
Los Angeles, California • Weston, Florida • Chicago, Illinois + 4 more
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Danicopan
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
7
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 4 more