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ClinicalTrials.gov public records Last synced Oct 10, 2026, 1:41 PM EDT

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Showing 49–67 of 67 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Paroxysmal Nocturnal Hemoglobinuria (PNH) Clear all
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 2, 2017 · Synced Oct 10, 2026, 1:41 PM EDT
Not listed No phase listed Observational

Identifying Characteristics of Bone Marrow Failure Syndromes

NCT00315419
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 21, 2010 · Synced Oct 10, 2026, 1:41 PM 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 13, 2009 · Synced Oct 10, 2026, 1:41 PM EDT
Completed Phase 4 Interventional Results available

Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT00867932
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 31, 2018 · Synced Oct 10, 2026, 1:41 PM EDT
Recruiting Phase 2 Interventional

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.

NCT06412497
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 10, 2026, 1:41 PM EDT
Completed Phase 3 Interventional Results available

Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

NCT04558918
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Oct 10, 2026, 1:41 PM EDT
Completed Not applicable Interventional

Nonmyeloablative Allogeneic Transplant

NCT01272817
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Nov 23, 2022 · Synced Oct 10, 2026, 1:41 PM EDT
Recruiting No phase listed Observational

Global PNH Patient Registry

NCT05755867
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 20, 2025 · Synced Oct 10, 2026, 1:41 PM 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 Sep 8, 2026 · Synced Oct 10, 2026, 1:41 PM 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 20, 2026 · Synced Oct 10, 2026, 1:41 PM EDT
Recruiting Phase 3 Interventional

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

NCT06934967
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 10, 2026, 1:41 PM EDT
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 4, 2025 · Synced Oct 10, 2026, 1:41 PM EDT
Completed Phase 2 Interventional Results available

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

NCT00566696
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 14, 2020 · Synced Oct 10, 2026, 1:41 PM EDT
Completed Phase 3 Interventional

Pegcetacoplan Long Term Safety and Efficacy Extension Study

NCT03531255
View directory record Official record
Conditions
PNH
Interventions
Pegcetacoplan
Drug
Lead sponsor
Apellis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
137 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Jacksonville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 10, 2026, 1:41 PM 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 17, 2026 · Synced Oct 10, 2026, 1:41 PM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 10, 2026, 1:41 PM 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 25, 2022 · Synced Oct 10, 2026, 1:41 PM 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 4, 2025 · Synced Oct 10, 2026, 1:41 PM 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 23, 2022 · Synced Oct 10, 2026, 1:41 PM EDT