Independent directory Public ClinicalTrials.gov records United States
Clinical Trials Finder
Objective public record navigation
Search Results

Search by objective public record fields.

Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.

Clear filters
ClinicalTrials.gov public records Last synced Jul 21, 2026, 1:46 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 67 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Paroxysmal Hemoglobinuria, Nocturnal Clear all
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
3
States / cities
New Brunswick, New Jersey • Philadelphia, Pennsylvania • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Jul 21, 2026, 1:46 PM 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 21, 2026, 1:46 PM 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 7, 2025 · Synced Jul 21, 2026, 1:46 PM EDT
Completed Phase 2 Interventional Results available

Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

NCT03030183
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Zilucoplan (RA101495)
Drug
Lead sponsor
Ra Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Duarte, California • Los Angeles, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 26, 2022 · Synced Jul 21, 2026, 1:46 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

NCT05133531
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Ravulizumab, Pozelimab, Cemdisiran, Eculizumab
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Whittier, California
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Jul 21, 2026, 1:46 PM EDT
Completed Phase 3 Interventional Results available

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

NCT03818607
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
ABP 959, Eculizumab
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 22, 2023 · Synced Jul 21, 2026, 1:46 PM 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 21, 2026, 1:46 PM 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 21, 2026, 1:46 PM EDT
Terminated Phase 1 Interventional

Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT01335165
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
ALXN1102, ALXN1103
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 21, 2014 · Synced Jul 21, 2026, 1:46 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 13, 2020 · Synced Jul 21, 2026, 1:46 PM 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 3, 2008 · Synced Jul 21, 2026, 1:46 PM EDT
Completed Phase 3 Interventional

Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

NCT00186823
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), 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
Source: ClinicalTrials.gov public record
Updated Jan 28, 2009 · Synced Jul 21, 2026, 1:46 PM EDT
Terminated Phase 2 Interventional Results available

Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study

NCT03225287
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Zilucoplan (RA101495)
Drug
Lead sponsor
Ra Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 27, 2023 · Synced Jul 21, 2026, 1:46 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 Jul 1, 2026 · Synced Jul 21, 2026, 1:46 PM 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 21, 2026, 1:46 PM EDT
Completed Phase 2 Interventional Results available

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

NCT00975975
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 25, 2016 · Synced Jul 21, 2026, 1:46 PM 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 21, 2026, 1:46 PM EDT
Completed Phase 3 Interventional Results available

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

NCT05630001
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jul 21, 2026, 1:46 PM 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 21, 2026, 1:46 PM 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 21, 2026, 1:46 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 20, 2010 · Synced Jul 21, 2026, 1:46 PM EDT
Completed Phase 3 Interventional Results available

A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria

NCT03406507
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Ravulizumab
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Atlanta, Georgia • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Mar 26, 2023 · Synced Jul 21, 2026, 1:46 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 30, 2018 · Synced Jul 21, 2026, 1:46 PM 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 3, 2008 · Synced Jul 21, 2026, 1:46 PM EDT