Clinical Trials on Paroxysmal Nocturnal Hemoglobinuria, Natural History Study

Total 129 results

NovelMed Therapeutics

Not yet recruiting

Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Paroxysmal Nocturnal Hemoglobinuria
Hoffmann-La Roche
Chugai Pharmaceutical

Active, not recruiting

A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. (COMMODORE 2)

Paroxysmal Nocturnal Hemoglobinuria

Spain, Korea, Republic of, France, Sweden, Thailand, Malaysia, China, Turkey, Netherlands, Japan, Australia, United Kingdom, Germany, South Africa, Brazil, Romania, Argentina, Poland, Greece, Hong Kong, Lithuania, Mexico, Philippines, Portuga... and more
Sanofi

Completed

A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults

Paroxysmal Nocturnal Hemoglobinuria

United States
Wuhan Createrna Science and Technology Co., Ltd

Completed

A Study of Single-dose MY008211A in Healthy Adults

Paroxysmal Nocturnal Hemoglobinuria

China
Regeneron Pharmaceuticals

Completed

Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Paroxysmal Nocturnal Hemoglobinuria

United Kingdom
Alexion Pharmaceuticals, Inc.

Available

Danicopan Early Access Program

Paroxysmal Nocturnal Hemoglobinuria | PNH | Extravascular Hemolysis
Alexion Pharmaceuticals, Inc.

Completed

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

Paroxysmal Nocturnal Hemoglobinuria

France, Italy, Spain, United States, Taiwan, Thailand, Korea, Republic of, Japan, Malaysia, Brazil, Germany, Israel, Czechia, Netherlands, Greece, United Kingdom, Canada, Poland
Asan Medical Center

Completed

Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome

Myelodysplastic Syndromes | Leukemia | Nonmalignant Neoplasm | Paroxysmal Nocturnal Hemoglobinuria

Korea, Republic of
Amgen

Completed

A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (DAHLIA)

Paroxysmal Nocturnal Hemoglobinuria

Spain, Italy, United States, Norway, Czechia, United Kingdom, Slovenia, Netherlands, Turkey, Portugal, Finland, France, Ireland, Sweden
Oregon Health and Science University

Completed

Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease

Sickle Cell Disease | Paroxysmal Nocturnal Hemoglobinuria | Rheologic Disease

United States
Institute of Hematology & Blood Diseases Hospital

Unknown

Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria (PNH-2012)

Urination Disorders | Thrombosis | Proteinuria | Anemia, Hemolytic | Hemoglobinuria, Paroxysmal | Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Bone Marrow Failure | Aplastic Anemia,

China
Institute of Hematology & Blood Diseases Hospital

Unknown

Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013)

Urination Disorders | Thrombosis | Proteinuria | Anemia, Hemolytic | Hemoglobinuria, Paroxysmal | Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Bone Marrow Failure | Aplastic Anemia,

China
St. Jude Children's Research Hospital

Completed

Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies

Lymphoma, Non-Hodgkin | Hodgkin Disease | Chronic Myeloid Leukemia | Acute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Myelodysplastic Syndrome (MDS) | Secondary AML | Juvenile Myelomonocytic Leukemia (JMML) | Secondary MDS

United States
Masonic Cancer Center, University of Minnesota

Not yet recruiting

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Acquired Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Acquired Amegakaryocytic Thrombocytopenia
Children's Hospital of Philadelphia

Recruiting

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

Paroxysmal Nocturnal Hemoglobinuria | Acquired Aplastic Anemia | Inherited Bone Marrow Failure Syndromes

United States
Alexion Pharmaceuticals, Inc.

Not yet recruiting

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

Pregnancy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Atypical Hemolytic Uremic Syndrome (aHUS) | Ultomiris-exposed Pregnant/ Postpartum | Generalized Myasthenia Gravis (gMG) | Neuromyelitis Optica Spectrum Disorder (NMOSD)
Lawson Health Research Institute
Alexion Pharmaceuticals, Inc.

Active, not recruiting

Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS

Transient Ischemic Attack | Paroxysmal Nocturnal Hemoglobinuria | Cerebral Vein Thrombosis | Embolic Stroke of Undetermined Source

Canada
St. Jude Children's Research Hospital

Completed

Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies

Acute Myeloid Leukemia | Leukemia | Hodgkin's Lymphoma | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Non-Hodgkin Lymphoma | Chronic Myeloid Leukemia | Myelodysplastic Syndrome | Paroxysmal Nocturnal Hemoglobinuria

United States
St. Jude Children's Research Hospital

Completed

Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies

Hodgkin Lymphoma | Myelodysplastic Syndrome (MDS) | Leukemia, Acute Lymphocytic (ALL) | Juvenile Myelomonocytic Leukemia (JMML) | Leukemia, Myeloid, Acute(AML) | Leukemia, Myeloid, Chronic(CML) | Hemoglobinuria, Paroxysmal Nocturnal (PNH) | Lymphoma, Non-Hodgkin (NHL)

United States
St. Jude Children's Research Hospital

Completed

Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

Myelodysplastic Syndrome (MDS) | Leukemia, Acute Lymphocytic (ALL) | Leukemia, Myeloid, Acute(AML) | Leukemia, Myeloid, Chronic(CML) | Hemoglobinuria, Paroxysmal Nocturnal (PNH) | Lymphoma, Non-Hodgkin (NHL) | Juvenile Myelomonocytic Leukemia(JMML)

United States
Scripps Health

Completed

Unrelated Donor Stem Cell Transplantation

Myelodysplastic Syndromes | Multiple Myeloma | Hodgkin's Lymphoma | Non-Hodgkin's Lymphoma | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Acute Myelogenous Leukemia | Chronic Myelogenous Leukemia | Small Lymphocytic Lymphoma | Paroxysmal Nocturnal Hemoglobinuria | Severe Aplastic Anemia | Myeloproliferative... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Recruiting

Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Congenital Amegakaryocytic Thrombocytopenia | Paroxysmal Nocturnal Hemoglobinuria | Shwachman-Diamond Syndrome | Bone Marrow Failure Syndrome | Congenital Pure Red Cell Aplasia | Hereditary Sideroblastic Anemia | Hematologic Neoplasm With Germline GATA2 Mutation | Hematologic Neoplasm With Germline... and other conditions

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sickle Cell Disease | Severe Congenital Neutropenia | Paroxysmal Nocturnal Hemoglobinuria | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Diamond Blackfan Anemia | Transfusion Dependent Alpha- or Beta- Thalassemia | Non-Malignant Hematologic Disorders

United States
Scripps Health

Completed

Nonmyeloablative Allogeneic Transplant (Mini-allo)

Melanoma | Sarcoma | Renal Cell Carcinoma | Breast Cancer | Multiple Myeloma | Hodgkin's Lymphoma | Non-Hodgkin's Lymphoma | Ovarian Cancer | Thymoma | Waldenstrom Macroglobulinemia | Mantle Cell Lymphoma | Chronic Lymphocytic Leukemia | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Chronic Myelogenous Leukemia and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Completed

Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders

Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Myeloproliferative Neoplasm | Paroxysmal Nocturnal Hemoglobinuria | de Novo Myelodysplastic Syndrome | Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative | Refractory Anemia | Refractory Anemia With... and other conditions

United States
National Cancer Institute (NCI)

Completed

Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer

Thrombocytopenia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid 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(16;16)(p13... and other conditions

United States
Roswell Park Cancer Institute

Active, not recruiting

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Acute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditions

United States
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States
Roswell Park Cancer Institute
National Cancer Institute (NCI)

Completed

Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders

Primary Myelofibrosis | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent... and other conditions

United States

Clinical Trials on Paroxysmal Nocturnal Hemoglobinuria, Natural History Study

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