73 completed, 12 recruiting, 11 terminated, 7 active not recruiting. 33 Phase 2, 29 Phase 3, 22 Phase 1, 10 Phase 4, 2 Not applicable.
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 |
|---|---|---|---|---|---|---|
| Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor Levels 5-40%) Joint Ultrasound (HEAD-US), Blood Sample for Coagulation Factor Measurement - Mild Hemophilia | Not applicable | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 150 | 2027-02-01 | 2026-08-17 |
| Pacritinib Effectiveness in Real-world Settings Not applicable- observational study - Myelofibrosis (MF) | Recruiting | Swedish Orphan Biovitrum | 60 | 2026-08-13 | 2026-10-21 | |
| Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study Pegcetacoplan - C3 Glomerulopathy (C3G), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) | Phase 4 | Recruiting | Swedish Orphan Biovitrum | 150 | 2026-08-05 | 2026-09-09 |
| A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment Avatrombopag 20 mg Oral Tablet - Aplastic Anemia | Phase 2 / Phase 3 | Not yet recruiting | Swedish Orphan Biovitrum | 26 | 2026-07-31 | 2026-08-26 |
| Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study Emapalumab - Dermatomyositis, Dermatomyositis Sine Myositis | Phase 2 | Recruiting | University of Miami | 5 | 2026-07-23 | 2026-08-25 |
| Pacritinib For Bone Marrow Fibrosis In Patients With Myelofibrosis Who Have Thrombocytopenia Pacritinib - Myelofibrosis，MF | Phase 2 | Recruiting | Grupo Español de Enfermedades Mieloproliferativas Crónicas PH Negativas | 30 | 2026-04-15 | 2026-10-02 |
| Pacritinib With Standard of Care Azacitidine or Decitabine as a Bridge to Allogeneic Hematopoietic Stem Cell Transplant for Patients With Accelerated and Blast Phase Myeloproliferative Neoplasms Pacritinib, Decitabine, Decitabine and Cedazuridine - Accelerated Phase Myeloproliferative Neoplasm, Blast Phase Myeloproliferative Neoplasm | Phase 2 | Recruiting | University of Washington | 27 | 2026-03-02 | 2026-08-19 |
| A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF Anakinra - Familial Mediterranean Fever (FMF ) | Phase 4 | Withdrawn | Swedish Orphan Biovitrum | 2026-03-01 | 2026-06-29 | |
| An Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) and Adult-Onset Still's Disease (AOSD) Anakinra (Kineret®) - Systemic Juvenile Idiopathic Arthritis (sJIA), Adult Onset Still's Disease (AOSD) | Phase 4 | Withdrawn | Swedish Orphan Biovitrum | 2025-09-01 | 2026-07-16 | |
| A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients Nitisinone - Hereditary Tyrosinemia, Type I | Withdrawn | Swedish Orphan Biovitrum | 2025-09-01 | 2025-12-23 | ||
| Assessment of Macrophage Activation syndromE in STill's Disease in Italy Macrophage Activation Syndrome (MAS), Still Disease, Juvenile Onset | Completed | Swedish Orphan Biovitrum | 32 | 2025-06-06 | 2025-12-02 | |
| A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) Pacritinib, Placebo - VEXAS, VEXAS Syndrome | Phase 2 | Recruiting | Swedish Orphan Biovitrum | 156 | 2025-05-28 | 2026-10-16 |
| Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa Efanesoctocog alfa - Hemophilia A | Phase 4 | Active not recruiting | Swedish Orphan Biovitrum | 252 | 2025-05-21 | 2026-09-30 |
| Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa Efanesoctocog alfa - Haemophilia A (Moderate or Severe) | Phase 3 | Active not recruiting | Swedish Orphan Biovitrum | 89 | 2025-03-06 | 2026-10-19 |
| Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A Efanesoctocog alfa - Hemophilia A | Phase 1 | Completed | Swedish Orphan Biovitrum | 24 | 2025-02-24 | 2026-06-25 |
| Pacritinib in Vacuoles, E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic (VEXAS) Syndrome Pacritinib - E1 Ubiqutin-activating Enzyme, X-linked, Autoinflammatory, Somatic Syndrome, VEXAS | Phase 1 | Recruiting | Washington University School of Medicine | 15 | 2025-02-13 | 2026-07-15 |
| A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis Efanesoctocog alfa - Haemophilia A (Moderate or Severe), Hemophilia, Classic | Phase 4 | Active not recruiting | Swedish Orphan Biovitrum | 37 | 2024-12-19 | 2026-04-30 |
| Emapalumab Prevention of CAR-T Cell Associated Toxicities Emapalumab, Cyclophosphamide, Fludarabine Phosphate - Large B-cell Lymphoma, Relapsed Non-Hodgkin Lymphoma | Phase 2 | Recruiting | Marcela V. Maus, M.D.,Ph.D. | 28 | 2024-09-18 | 2026-10-06 |
| Prevalence of Synovitis in Patients With Haemophilia A Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Haemophilia joint health score (HJHS) - Haemophilia A, Synovitis | Recruiting | University Hospital, Bonn | 300 | 2024-04-15 | 2024-04-16 | |
| Avatrombopag vs. Placebo for CIT in GI Malignancies Avatrombopag, Matching Placebo - Gastrointestinal Cancer, Gastrointestinal Neoplasms | Phase 2 | Completed | Hanny Al-Samkari, MD | 49 | 2023-11-01 | 2026-08-31 |
Showing 20 of 113. Sign in to see all of them.
Hemophilia A (22) · Hemophilia B (14) · Blood Coagulation Disorders (10) · Coagulation Protein Disorders (10) · Arthritis, Juvenile (8) · Lymphohistiocytosis, Hemophagocytic (8) · Stomatitis (7) · Hemorrhagic Disorders (6) · Still's Disease, Adult-Onset (6) · Genetic Diseases, Inborn (5) · Hematologic Diseases (5) · Leukemia (4) · Macrophage Activation Syndrome (4) · Neoplasms (4) · Tyrosinemias (4)
Interleukin 1 Receptor Antagonist Protein (17) · Emapalumab (13) · Fibroblast Growth Factor 7 (13) · factor VIII-Fc fusion protein (10) · factor IX Fc fusion protein (7) · nitisinone (7) · Factor VIII (6) · 11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene (5) · Blood Specimen Collection (4) · Radiotherapy (4) · pegcetacoplan (4) · BIVV001 (3) · Cyclophosphamide (3) · Whole-Body Irradiation (3) · avatrombopag (3)
United States (47) · Italy (37) · Germany (35) · France (34) · United Kingdom (33) · Spain (30) · Sweden (19) · Netherlands (16) · Canada (15) · Belgium (14) · Czechia (13) · Australia (11) · Ireland (11) · Poland (11) · Japan (9)
Erik Brouwer, MD (4) · Kristina Timdahl, MD (4) · Elena Santagostino, MD (3) · Paul Harmatz, MD (2) · Alice J. Cohen, MD (1) · Amy D Shapiro, MD (1) · Andrea Vambutas, MD (1)
PMID 25222806, PMID 38662147, PMID 25880826, PMID 32374962, PMID 33719084, PMID 35835061, PMID 36848635, PMID 37001971, PMID 39091324, PMID 17896890, PMID 21199898, PMID 22110246, PMID 22915649, PMID 24227821, PMID 24304002, PMID 25133431, PMID 25196897, PMID 25912075, PMID 27244221, PMID 28159192, PMID 31152037, PMID 32779863, PMID 33232476, PMID 33605029, PMID 33935163, PMID 34242387, PMID 35105709, PMID 35421219, PMID 35460313, PMID 36735911, PMID 38266154, PMID 38907160, PMID 38922990, PMID 39121163, PMID 39583653, PMID 39872008, PMID 40099856, PMID 40289300, PMID 40616274, PMID 41753113
2014 (2), 2017 (2), 2023 (2), 2025 (2), 2009 (1), 2016 (1)