| Withdrawn | A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients Hereditary Tyrosinemia, Type I | — | 2025-09-01 |
| Not Yet Recruiting | A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF Familial Mediterranean Fever (FMF ) | Phase 4 | 2025-09-01 |
| Completed | Assessment of Macrophage Activation syndromE in STill's Disease in Italy Macrophage Activation Syndrome (MAS), Still Disease, Juvenile Onset, Still's Disease, Adult-Onset | — | 2025-06-06 |
| Recruiting | A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) VEXAS, VEXAS Syndrome | Phase 2 | 2025-05-28 |
| Recruiting | Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatme Hemophilia A | Phase 4 | 2025-05-21 |
| Recruiting | Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alf Severe Haemophilia A | Phase 3 | 2025-03-06 |
| Recruiting | Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haem Hemophilia A | Phase 1 | 2025-02-24 |
| Active Not Recruiting | A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia | Phase 4 | 2024-12-19 |
| Completed | Assessment of Macrophage Activation syndromE in STill's Disease Macrophage Activation Syndrome, Still's Disease, Adult-Onset, Stills Disease, Juvenile-Onset | — | 2023-09-27 |
| Terminated | An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis Wit Hemophilia A, Hemophilia B | Phase 4 | 2023-08-24 |
| Active Not Recruiting | A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Hemophilia A, Severe | Phase 3 | 2023-07-31 |
| Recruiting | A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria | — | 2023-06-26 |
| Completed | A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Trea Primary Hemophagocytic Lymphohistiocytosis | Phase 4 | 2023-02-03 |
| Completed | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD) Cold Agglutinin Disease | Phase 3 | 2022-10-20 |
| Active Not Recruiting | A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD) Still's Disease, Juvenile Onset, Still's Disease, Adult-Onset | Phase 3 | 2022-08-24 |
| Completed | An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP Immune Thrombocytopenia, ITP | — | 2022-02-22 |
| Completed | A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoieti Transplant-Associated Thrombotic Microangiopathy | Phase 2 | 2022-02-01 |
| Completed | Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease | Phase 3 | 2021-12-15 |
| Terminated | Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Graft Failure | Phase 2 | 2021-05-25 |
| Completed | Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST) Hemophilia A, Hemophilia B | — | 2021-05-05 |
| Completed | A Study to Evaluate Emapalumab in Japanese Healthy Volunteers. Rare Diseases | Phase 1 | 2021-01-14 |
| Completed | A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy II Chronic Gout | Phase 3 | 2020-11-30 |
| Terminated | A Clinical Study to Investigate Interferon Gamma (IFNɣ) Signature in Patients Post HSCT and in Patients With I Graft Failure | — | 2020-11-16 |
| Completed | A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy Chronic Gout | Phase 3 | 2020-08-18 |
| Terminated | A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH Hemophagocytic Lymphohistiocytoses | Phase 2 / Phase 3 | 2020-06-02 |
| Terminated | Efficacy and Safety of Emapalumab and Anakinra in Reducing Hyperinflammation and Respiratory Distress in Patie SARS-CoV-2 | Phase 2 / Phase 3 | 2020-04-02 |
| Active Not Recruiting | A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health Hemophilia A | — | 2020-03-30 |
| Completed | Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint Examination Hemophilia A | N/A | 2020-01-13 |
| Completed | An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemoph Hemophilia B | — | 2019-09-12 |
| Completed | Long-term Safety Study of Kineret® in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) Still Disease, Juvenile Onset | — | 2019-04-10 |
| Completed | Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis Primary Hemophagocytic Lymphohistiocytosis | Phase 3 | 2019-02-06 |
| Completed | A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Sanfilippo Syndrome Type A (MPS IIIA) | Phase 1 / Phase 2 | 2019-01-19 |
| Completed | rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A Hemophilia A With Inhibitor | — | 2018-11-07 |
| Completed | Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophili Haemophilia A, Haemophilia B | — | 2018-10-30 |
| Completed | A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in Franc Haemophilia B | — | 2018-09-12 |
| Completed | A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients Sanfilippo Syndrome Type A (MPS IIIA) | Phase 1 / Phase 2 | 2018-06-19 |
| Completed | A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic, Arthritis, Juvenile | Phase 2 | 2018-02-20 |
| Terminated | A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD) Still's Disease, Adult-Onset, Still's Disease, Juvenile-Onset | Phase 3 | 2017-09-26 |
| Completed | Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Fail Hemophilia A | Phase 4 | 2017-08-29 |
| Recruiting | A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis | Phase 3 | 2017-06-26 |
| Completed | A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia Haemophilia A, Haemophilia B | — | 2017-05-09 |
| Completed | A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drug Drug Drug Interaction | Phase 1 | 2017-03-28 |
| Completed | Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis Hemophagocytic Lymphohistiocytoses | — | 2016-12-14 |
| Completed | Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Hemophilia A | — | 2016-12-01 |
| Completed | A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Acute Gouty Arthritis Acute Gouty Arthritis | Phase 2 | 2016-12-01 |
| Completed | Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohist Hemophagocytic Lymphohistiocytosis | — | 2016-10-13 |
| Completed | Kineret CAPS Post Authorisation Study Cryopyrin-Associated Periodic Syndromes | — | 2015-04-24 |
| Completed | Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 Hereditary Tyrosinemia, Type I | Phase 3 | 2014-12-01 |
| Completed | A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatmen Hemophagocytic Lymphohistiocytosis | Phase 2 / Phase 3 | 2014-08-04 |
| Completed | A Non-interventional Retrospective Study of the Current Treatment Practice in European Haemophilia Care Haemophilia A, Haemophilia B | — | 2014-03-01 |
| Terminated | Safety and Tolerability of SOBI002 in Healthy Volunteers Following Single and Repeated Administration Healthy | Phase 1 | 2013-12-01 |
| Completed | Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care Hereditary Tyrosinemia, Type I | — | 2013-09-01 |
| Completed | Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohisti Primary Haemophagocytic Lymphohistiocytosis | Phase 2 / Phase 3 | 2013-07-01 |
| Completed | Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules. Healthy | Phase 1 | 2013-05-01 |
| Completed | Kineret in the Treatment of Rheumatoid Arthritis Rheumatoid Arthritis | — | 2012-10-01 |
| Completed | Taste and Palatability of Orfadin Suspension Hereditary Tyrosinemia, Type I | Phase 1 | 2012-10-01 |
| Completed | Bioequivalence of Orfadin Suspension Compared to Orfadin Capsules, and the Effect of Food on the Bioavailabili Healthy Volunteers | Phase 1 | 2012-08-01 |
| Completed | First in Human Study of an Anti-IFN Gamma Monoclonal Antibody in Healthy Volunteers Healthy Volunteers | Phase 1 | 2011-09-01 |
| Terminated | Comparison of rhBSSL With Placebo When Added to Infant Formula or Pasteurized Breast Milk During 4 Weeks of Tr Prevention of Growth Restriction | Phase 3 | 2011-05-01 |
| Completed | Characterization of Cyanocobalamin in Healthy Voluteers After Intranasal and Intramuscual Administration Focus: Bioavailability | Phase 1 | 2011-02-01 |
| Completed | Study to Characterize the Effect of Heparin on Palifermin Activity Oral Mucositis | Phase 1 | 2010-07-01 |
| Completed | Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency Cystic Fibrosis, Exocrine Pancreatic Insufficiency | Phase 2 | 2008-08-01 |
| Completed | Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Infant Formula for Preterm Replacement Therapy in Preterm Infants | Phase 2 | 2008-04-01 |
| Completed | Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Pasteurized Breast Milk fo Replacement Therapy in Preterm Infants | Phase 2 | 2008-03-01 |
| Withdrawn | A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Locally Advanced Head and Neck Canc Head and Neck Cancer | Phase 1 / Phase 2 | 2007-10-01 |
| Completed | Efficacy and Tolerability of Novel A2A Agonist in Treatment of Diabetic Neuropathic Pain Diabetic Neuropathic Pain | Phase 2 | 2007-05-01 |
| Completed | A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Multiple Myeloma Multiple Myeloma | Phase 3 | 2006-12-01 |
| Completed | A Phase 1 Dose-escalation Study to Evaluate the Safety and Pharmacokinetics (PK) of Palifermin in Subjects Wit Leukemia | Phase 1 | 2006-08-01 |
| Completed | PROMPT - Palifermin in Reduction of Oral Mucositis in PBSC Transplantation Non-Hodgkin's Lymphoma, Multiple Myeloma | Phase 4 | 2006-04-01 |
| Completed | Trial to Evaluate Palifermin in the Reduction of Acute Graft Versus Host Disease in Patients With Hematologic Graft Versus Host Disease, Hematologic Malignancies | Phase 2 | 2005-12-01 |
| Completed | Palifermin DDI (Drug Drug Interaction) Healthy Volunteers | Phase 1 | 2005-12-01 |
| Completed | Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer Mucositis, Solid Tumors, Stomatitis | Phase 3 | 2005-08-01 |
| Completed | A Study of Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Canc Mucositis, Head and Neck Cancer | Phase 1 / Phase 2 | 2005-07-01 |
| Completed | Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE) Cancer, Lymphoma, Leukemia | Phase 3 | 2005-01-01 |
| Completed | Study to Evaluate Palifermin in the Reduction of Dysphagia in Patients With Locally Advanced Non-Small Cell Lu Dysphagia, Non-Small Cell Lung Cancer, Lung Cancer | Phase 2 | 2005-01-01 |
| Completed | Pharmacokinetic Study of Palifermin in Subjects Receiving Radiation Therapy and Chemotherapy Followed by Blood Leukemia, Lymphoma, Myeloma | Phase 1 | 2001-12-01 |