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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: PNH Clear all
Terminated Phase 2 Interventional Results available

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

NCT03333486
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Jul 22, 2026, 8:29 AM EDT
Completed No phase listed Observational

Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study

NCT01412047
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 17, 2017 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 2 Interventional Results available

Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

NCT03472885
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Danicopan, Eculizumab
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
2
States / cities
Baltimore, Maryland • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 11, 2023 · Synced Jul 22, 2026, 8:29 AM EDT
Active, not recruiting Not applicable Interventional

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

NCT03047746
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Recruiting Phase 2 Interventional

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

NCT03520647
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional

Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria

NCT00130000
View directory record Official record
Conditions
Paroxysmal Hemoglobinuria, Nocturnal
Interventions
Eculizumab
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 22, 2026, 8:29 AM EDT
Active, not recruiting Phase 3 Interventional

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

NCT05389449
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Not applicable Interventional

Unrelated Donor Stem Cell Transplantation

NCT01364363
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 22, 2022 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional Results available

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

NCT00122317
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 12, 2018 · Synced Jul 22, 2026, 8:29 AM EDT
Recruiting No phase listed Observational

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

NCT06312644
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional Results available

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT02946463
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 13, 2024 · Synced Jul 22, 2026, 8:29 AM EDT
Active, not recruiting No phase listed Observational

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

NCT07413679
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional

Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

NCT00122330
View directory record Official record
Conditions
Hemoglobinuria, Paroxysmal
Interventions
eculizumab
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
16
States / cities
Duarte, California • Los Angeles, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2006 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 2 Interventional Results available

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

NCT00397813
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 30, 2020 · Synced Jul 22, 2026, 8:29 AM EDT
Recruiting No phase listed Observational

Home Reported Outcomes in PNH

NCT06411626
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 26, 2025 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 2 Interventional

Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies

NCT00145613
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 12, 2009 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional Results available

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

NCT04469465
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 3, 2025 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT03500549
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 24, 2022 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 2 Interventional

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

NCT04965597
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 2 Interventional Results available

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

NCT03427060
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 3, 2025 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 2 Interventional

Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies

NCT00143559
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 28, 2009 · Synced Jul 22, 2026, 8:29 AM EDT
Active, not recruiting No phase listed Observational

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

NCT07413250
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed No phase listed Observational

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

NCT01374360
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced Jul 22, 2026, 8:29 AM EDT
Completed Phase 3 Interventional

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

NCT00122304
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 20, 2007 · Synced Jul 22, 2026, 8:29 AM EDT