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ClinicalTrials.gov public records Last synced Oct 8, 2026, 10:06 AM EDT

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Showing 49–67 of 67 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Paroxysmal Hemoglobinuria, Nocturnal Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

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

NCT06326814
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 22, 2024 · Synced Oct 8, 2026, 10:06 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

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

NCT01294891
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced Oct 8, 2026, 10:06 AM EDT
Recruiting Phase 2 Interventional

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

NCT01174108
View directory record Official record
Conditions
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Interventions
Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
Device · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
2
States / cities
Baltimore, Maryland • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 8, 2026, 10:06 AM EDT
Recruiting Phase 2 Interventional

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT04901936
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
Interventions
Pegcetacoplan
Drug
Lead sponsor
Apellis Pharmaceuticals, Inc.
Industry
Eligibility
12 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jun 26, 2026 · Synced Oct 8, 2026, 10:06 AM EDT
Completed Phase 2 Interventional Results available

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

NCT00004143
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 5, 2014 · Synced Oct 8, 2026, 10:06 AM 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 8, 2026, 10:06 AM EDT
Completed Phase 2 Interventional Results available

Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders

NCT00587054
View directory record Official record
Conditions
Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years to 55 Years
Enrollment
129 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2011
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 10, 2017 · Synced Oct 8, 2026, 10:06 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 Sep 8, 2026 · Synced Oct 8, 2026, 10:06 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 4, 2006 · Synced Oct 8, 2026, 10:06 AM 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 8, 2026, 10:06 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 4, 2025 · Synced Oct 8, 2026, 10:06 AM EDT
Terminated Phase 3 Interventional Results available

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

NCT05131204
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 8, 2025 · Synced Oct 8, 2026, 10:06 AM EDT
Recruiting No phase listed Observational

The International PNH Interest Group PNH Registry

NCT06524726
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 29, 2024 · Synced Oct 8, 2026, 10:06 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 17, 2026 · Synced Oct 8, 2026, 10:06 AM EDT
Completed No phase listed Observational

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

NCT00039923
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 8, 2026, 10:06 AM 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 8, 2026, 10:06 AM EDT
Completed Phase 3 Interventional Accepts healthy volunteers

Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria

NCT00098280
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 8, 2026, 10:06 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 23, 2022 · Synced Oct 8, 2026, 10:06 AM EDT
Active, not recruiting Phase 3 Interventional

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

NCT04747613
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Iptacopan
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 100 Years
Enrollment
208 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
11
States / cities
Duarte, California • Los Angeles, California • Orange, California + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 7, 2026 · Synced Oct 8, 2026, 10:06 AM EDT