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 come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.

Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 6, 2026, 2:51 AM EDT

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

Showing 25–48 of 67 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Paroxysmal Hemoglobinuria, Nocturnal Clear all
Active, not recruiting Phase 3 Interventional

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

NCT04432584
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria
Interventions
Crovalimab, Eculizumab
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years and older
Enrollment
190 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Charlotte, North Carolina • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 6, 2026, 2:51 AM 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 30, 2026 · Synced Oct 6, 2026, 2:51 AM EDT
Completed Phase 1 Interventional Results available

A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH

NCT02264639
View directory record Official record
Conditions
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions
Pegcetacoplan
Drug
Lead sponsor
Apellis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
7
States / cities
Los Angeles, California • Miami Lakes, Florida • Louisville, Kentucky + 4 more
Source: ClinicalTrials.gov public record
Updated Jan 8, 2021 · Synced Oct 6, 2026, 2:51 AM 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 13, 2026 · Synced Oct 6, 2026, 2:51 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 6, 2026, 2:51 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 6, 2026, 2:51 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 6, 2026, 2:51 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

First-In-Human Clinical Study of the C3 Complement Inhibitor AMY- 101 in Healthy Male Volunteers

NCT03316521
View directory record Official record
Conditions
Complement Mediated Diseases
Interventions
AMY-101
Drug
Lead sponsor
Amyndas Pharmaceuticals S.A.
Industry
Eligibility
18 Years to 60 Years · Male only
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017
U.S. locations
1
States / cities
High Point, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 16, 2018 · Synced Oct 6, 2026, 2:51 AM 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 6, 2026, 2:51 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 6, 2026, 2:51 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 17, 2026 · Synced Oct 6, 2026, 2:51 AM EDT
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 6, 2026, 2:51 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 12, 2023 · Synced Oct 6, 2026, 2:51 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 6, 2026, 2:51 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 17, 2026 · Synced Oct 6, 2026, 2:51 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 18, 2017 · Synced Oct 6, 2026, 2:51 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 6, 2026, 2:51 AM 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 6, 2026, 2:51 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 9, 2026 · Synced Oct 6, 2026, 2:51 AM 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 29, 2009 · Synced Oct 6, 2026, 2:51 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 29, 2009 · Synced Oct 6, 2026, 2:51 AM 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 23, 2023 · Synced Oct 6, 2026, 2:51 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 6, 2026, 2:51 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 6, 2026, 2:51 AM EDT