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Conditions: Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia(JMML), Hemoglobinuria, Paroxysmal Nocturnal (PNH), Lymphoma, Non-Hodgkin (NHL), Myelodysplastic Syndrome (MDS)
Interventions: Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows:; Device · Procedure · Drug
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 2 Years to 21 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2009
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions: Coversin; Drug
Lead sponsor: AKARI Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 1
States / cities: New York, New York

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 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: 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

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: Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Hodgkin's Disease, Multiple Myeloma, Myelofibrosis, Anemia, Aplastic, Hemoglobinuria, Paroxysmal
Interventions: Basiliximab; Drug
Lead sponsor: Indiana University; Other
Eligibility: 18 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 1
States / cities: Indianapolis, Indiana

Conditions: Paroxysmal Nocturnal Hemoglobinuria
Interventions: Not listed
Lead sponsor: International PNH Interest Group; Other
Eligibility: Not listed
Enrollment: 2,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Altamonte Springs, Florida

Conditions: Paroxysmal Hemoglobinuria, Nocturnal
Interventions: eculizumab; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 187 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 14
States / cities: Stanford, California • Hartford, Connecticut • Weston, Florida + 10 more

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: 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: 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: Paroxysmal Nocturnal Hemoglobinuria
Interventions: Not listed
Lead sponsor: Aplastic Anemia and MDS International Foundation; Other
Eligibility: Not listed
Enrollment: 500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2031
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
Interventions: Rituximab, Rabbit ATG, Cyclophosphamide, Fludarabine, Total Body Irradiation, Cell Infusion, Post-Transplant G-CSF, Tacrolimus, Mycophenolate Mofetil; Drug · Radiation · Biological
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: 0 Years to 75 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2036
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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: 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: 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: Hemoglobinuria, Paroxysmal
Interventions: eculizumab; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 85 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 19
States / cities: Los Angeles, California • Stanford, California • Hartford, Connecticut + 15 more

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, 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: Paroxysmal Nocturnal Hemoglobinuria
Interventions: PNH-relevant therapies; Other
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years to 99 Years
Enrollment: 128 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: East Hanover, New Jersey

Conditions: Hemoglobinuria, Paroxysmal
Interventions: Eculizumab; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 75 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Paroxysmal Nocturnal Hemoglobinuria, PNH
Interventions: Danicopan; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Boston, Massachusetts

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

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Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer

Transfer of GPI-Linked Proteins to Transfused Patients With Paroxysmal Nocturnal Hemoglobinuria

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer

The International PNH Interest Group PNH Registry

Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

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

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

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

Global PNH Patient Registry

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.

Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors

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

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

Home Reported Outcomes in PNH

Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria

Long-term Safety of Danicopan: IPIG Registry-based Cohort Study

Molecular and Clinical Analysis of Bone Marrow Failure: A Secondary Research Study