10 completed, 2 unknown, 1 enrolling by invitation, 1 recruiting. 6 Phase 2, 2 Phase 1, 2 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Completed 4, Unknown 2 |
| Phase 1 | 2 | Completed 2 |
| Phase 3 | 2 | Completed 2 |
Follow Hemoglobinuria: new trials by email Literature Review of Hemoglobinuria trials Research Report on Hemoglobinuria
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea Hemoglobinuria, Paroxysmal | Enrolling by invitation | Yonsei University | 78 | 2026-07-22 | 2026-11-06 | |
| The International PNH Interest Group PNH Registry Paroxysmal Nocturnal Hemoglobinuria | Recruiting | International PNH Interest Group | 2000 | 2024-05-10 | 2024-07-29 | |
| A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH Pegcetacoplan - PNH | Phase 2 | Completed | Apellis Pharmaceuticals, Inc. | 4 | 2018-08-16 | 2020-12-22 |
| Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH Elizaria®, Soliris® - Paroxysmal Nocturnal Hemoglobinuria, Marchiafava-Micheli Syndrome | Phase 3 | Completed | AO GENERIUM | 32 | 2017-11-29 | 2021-09-01 |
| A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) ACH-0144471 - Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 8 | 2017-06-22 | 2023-03-14 |
| Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Danicopan - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 10 | 2017-03-31 | 2022-06-23 |
| Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. Pegcetacoplan - Paroxysmal Nocturnal Hemoglobinuria | Phase 1 | Completed | Apellis Pharmaceuticals, Inc. | 23 | 2015-12-01 | 2021-01-11 |
| A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH Pegcetacoplan - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 1 | Completed | Apellis Pharmaceuticals, Inc. | 9 | 2015-02-23 | 2021-01-08 |
| Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids - Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 50 | 2013-01-01 | 2013-01-03 |
| Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids - Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria | Phase 2 | Unknown | Institute of Hematology & Blood Diseases Hospital, China | 120 | 2012-07-01 | 2013-01-03 |
| Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes Hemoglobinuria | Completed | Alexion Pharmaceuticals, Inc. | 5580 | 2010-09-01 | 2026-06-24 | |
| Incidence of Rhabdomyolysis Among Patients Admitted With Jimsonweed Ingestions Rhabdomyolysis, Myoglobinuria | Completed | Akron Children's Hospital | 46 | 2008-01-01 | 2008-07-28 | |
| Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies CliniMACS, Stem cell transplantation, Fludarabine - Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML) | Phase 2 | Completed | St. Jude Children's Research Hospital | 73 | 2007-12-14 | 2020-04-14 |
| Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows: - Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML) | Phase 3 | Completed | St. Jude Children's Research Hospital | 57 | 2002-03-01 | 2009-01-29 |
Hemoglobinuria, Paroxysmal (12) · Anemia (7) · Hematologic Diseases (7) · Anemia, Hemolytic (4) · Hemolysis (4) · Myelodysplastic Syndromes (4) · Anemia, Aplastic (3) · Bone Marrow Failure Disorders (3) · Proteinuria (3) · Bone Marrow Diseases (2) · Leukemia (2) · Leukemia, Myeloid, Acute (2) · Leukemia, Myelomonocytic, Juvenile (2) · Lymphoma (2)
Female Urogenital Diseases (14) · Female Urogenital Diseases and Pregnancy Complications (14) · Male Urogenital Diseases (14) · Pathological Conditions, Signs and Symptoms (14) · Signs and Symptoms (14) · Urogenital Diseases (14) · Urologic Diseases (14) · Urological Manifestations (14) · Hemic and Lymphatic Diseases (12) · Urination Disorders (12) · Proteinuria (11) · Bone Marrow Diseases (10) · Anemia, Hemolytic (8) · Myelodysplastic Syndromes (8) · Anemia (5)
pegcetacoplan (3) · Cyclosporine (2) · Glucocorticoids (2) · Levamisole (2) · Stem Cell Transplantation (2) · danicopan (2) · Alemtuzumab (1) · Antibodies (1) · Antilymphocyte Serum (1) · Cyclophosphamide (1) · Filgrastim (1) · Granulocyte Colony-Stimulating Factor (1) · Melphalan (1) · Muromonab-CD3 (1) · Mycophenolic Acid (1)
Alexion Pharmaceuticals, Inc. (3) · Apellis Pharmaceuticals, Inc. (3) · Institute of Hematology & Blood Diseases Hospital, China (2) · St. Jude Children's Research Hospital (2) · AO GENERIUM (1) · Akron Children's Hospital (1) · International PNH Interest Group (1) · Yonsei University (1)
United States (5) · New Zealand (3) · South Korea (3) · China (2) · Italy (2) · Bulgaria (1) · Hong Kong (1) · Malaysia (1) · Russia (1) · Serbia (1) · Thailand (1) · United Kingdom (1)
Federico Grossi, MD, PhD (3) · Brandon Triplett, MD (1) · Gregory A. Hale, M.D. (1) · Jeff Szer, MB BS FRACP (1) · Martha Blackford (1) · Oksana A. Markova, MD (1) · Richard Kelly, MBChB PhD (1)
PMID 35869170, PMID 33121236, PMID 34398258, PMID 39079163, PMID 41124654
2016 (1), 2017 (1)