| Withdrawn | Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia Hypophosphatasia | Phase 4 | 2026-07-29 |
| Recruiting | Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants Healthy Adult Participants | Phase 1 | 2026-03-26 |
| Recruiting | A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravu Generalized Myasthenia Gravis, gMG | Phase 4 | 2026-03-04 |
| Active Not Recruiting | Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Paroxysmal Nocturnal Hemoglobinuria, PNH | — | 2026-01-14 |
| Recruiting | Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS aHUS, Atypical Hemolytic Uremic Syndrome | Phase 4 | 2025-12-19 |
| Recruiting | Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Ass Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis | Phase 2 | 2025-11-26 |
| Recruiting | ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly Acromegaly | Phase 2 | 2025-10-28 |
| Recruiting | ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy BAG3 Mutation Associated Dilated Cardiomyopathy | Phase 1 / Phase 2 | 2025-10-24 |
| Recruiting | Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers Transthyretin-type Cardiac Amyloidosis | Phase 4 | 2025-10-01 |
| Recruiting | Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN) Primary Membranous Nephropathy | Phase 2 | 2025-09-19 |
| Recruiting | A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory A Relapsed AL Amyloidosis, Refractory AL Amyloidosis, Light Chain Amyloidosis | Phase 1 / Phase 2 | 2025-08-18 |
| Active Not Recruiting | Long-term Safety of Danicopan: IPIG Registry-based Cohort Study Paroxysmal Nocturnal Hemoglobinuria, PNH | — | 2025-08-18 |
| Recruiting | Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Hav Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis | Phase 3 | 2025-08-11 |
| Recruiting | Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis Generalized Myasthenia Gravis, Anti-AChR Antibody Positive | — | 2025-07-31 |
| Recruiting | Study of Ravulizumab in Pediatric Participants With Primary IgAN IgAN, IgAVN, Immunoglobulin A Nephropathy | Phase 3 | 2025-06-14 |
| Recruiting | Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravuliz Delayed Graft Function, DGF, Kidney Transplant | Phase 3 | 2025-05-19 |
| Recruiting | Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation Antibody-Mediated Rejection, Kidney Transplantation, Biopsy-proven Histologic Scores | Phase 2 | 2025-03-07 |
| Active Not Recruiting | Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD NMOSD, Neuromyelitis Optica Spectrum Disorders | Phase 3 | 2025-01-16 |
| Completed | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With Generalized Myasthenia Gravis (gMG), Refractory gMG | Phase 3 | 2025-01-06 |
| Recruiting | Study of Ultomiris® (Ravulizumab) Safety in Pregnancy Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH) | — | 2024-12-16 |
| Recruiting | PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Grav Generalized Myasthenia Gravis, gMG | Phase 3 | 2024-11-13 |
| Completed | PK Study of Gefurulimab SC in Healthy Chinese Adult Participants Healthy | Phase 1 | 2024-10-28 |
| Completed | Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Part Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 3 | 2024-10-10 |
| Active Not Recruiting | Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP Hypophosphatasia | Phase 3 | 2024-05-14 |
| Active Not Recruiting | Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa Hypophosphatasia | Phase 3 | 2024-04-02 |
| Recruiting | Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN) Immunoglobulin A Nephropathy, IgAN | Phase 3 | 2024-03-29 |
| Recruiting | Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies Neuromyelitis Optica Spectrum Disorder, NMOSD, AQP4+ NMOSD | — | 2024-02-01 |
| Completed | Potential Drug Interaction Study Between Vemircopan and Rosuvastatin, Metformin, Levonorgestrel/Ethinyl Estrad Healthy Participants | Phase 1 | 2024-01-17 |
| Active Not Recruiting | Study of ALXN2220 Versus Placebo in Adults With ATTR-CM Transthyretin Amyloid Cardiomyopathy | Phase 3 | 2024-01-11 |
| Completed | A Study to Evaluate Potential Drug Interactions Between ALXN2080 and Itraconazole, Fluconazole & Carbamazepine Healthy | Phase 1 | 2024-01-10 |
| Active Not Recruiting | Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Hypophosphatasia | Phase 3 | 2024-01-03 |
| Completed | A Study of Subcutaneous Gefurulimab Using Prefilled Syringe Versus Autoinjector in Healthy Adult Participants Healthy Adult Participants | Phase 1 | 2023-11-22 |
| Completed | Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China Atypical Hemolytic Uremic | Phase 3 | 2023-07-14 |
| Completed | Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | 2023-07-05 |
| Active Not Recruiting | Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Ge Generalized Myasthenia Gravis, gMG | Phase 3 | 2023-06-24 |
| Active Not Recruiting | Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism Chronic Hypoparathyroidism, Endocrine System Diseases, Parathyroid Diseases | Phase 3 | 2023-06-07 |
| Completed | Safety and Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN1920 in Healthy Participants Healthy Participants | Phase 1 | 2023-04-19 |
| Completed | A Study to Investigate the Potential Drug Interactions Between ALXN2080 and Rosuvastatin and Metformin in Heal Healthy | Phase 1 | 2023-04-12 |
| Active Not Recruiting | ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE Chronic Kidney Disease, CKD, Cardiac Disease | Phase 3 | 2023-04-06 |
| Completed | A Study to Investigate the Pharmacokinetic Profile of ALXN2050 Modified Release Prototype Formulations and Imm Healthy Participants | Phase 1 | 2023-03-15 |
| Terminated | Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of ALXN1820 in Adult Participants With Sickle Cel Sickle Cell Disease (SCD) | Phase 2 | 2023-02-22 |
| Completed | Potential Drug Interaction Between ALXN2040 and Rosuvastatin Healthy Participants | Phase 1 | 2023-02-01 |
| Active Not Recruiting | Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis Generalized Myasthenia Gravis | Phase 3 | 2022-11-21 |
| Active Not Recruiting | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | 2022-10-28 |
| Active Not Recruiting | A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 D Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM) | — | 2022-10-14 |
| Completed | Bioequivalence Between 180 mg and 60 mg ALXN2050 Tablets and Food Effect on ALXN2050 Pharmacokinetics Healthy Adult Participants | Phase 1 | 2022-10-13 |
| Completed | Safety and Tolerability, PK, and PD Study of Single and Multiple ALXN2080 Doses in Healthy Participants Healthy | Phase 1 | 2022-09-12 |
| Recruiting | A Prospective Sub-Study of the Global Hypophosphatasia Registry Hypophosphatasia | — | 2022-08-25 |
| Active Not Recruiting | Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants Healthy | Phase 1 | 2022-08-16 |
| Active Not Recruiting | Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy Methylmalonic Acidemia | — | 2022-07-15 |
| Completed | Safety and Pharmacokinetic Study of Subcutaneous ALXN1720 in Participants With Proteinuria Proteinuria | Phase 1 | 2022-06-29 |
| Recruiting | Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD Neuromyelitis Optica Spectrum Disorder | Phase 2 / Phase 3 | 2022-06-23 |
| Completed | Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria | — | 2022-06-17 |
| Terminated | Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis Generalized Myasthenia Gravis, Myasthenia Gravis | Phase 2 | 2022-04-27 |
| Completed | Safety and Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN1910 in Healthy Participants Healthy | Phase 1 | 2022-04-12 |
| Terminated | Study of ALXN2050 in Participants With Hepatic Impairment Impaired Hepatic Function, Healthy | Phase 1 | 2022-04-07 |
| Terminated | Study of ALXN2050 in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) Lupus Nephritis, Immunoglobulin A Nephropathy, IgAN | Phase 2 | 2022-01-14 |
| Completed | Drug-Drug Interaction (DDI) Study of ALXN2050 in Healthy Adult Participants Healthy | Phase 1 | 2022-01-11 |
| Withdrawn | Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia Warm Autoimmune Hemolytic Anemia | Phase 2 | 2022-01-01 |
| Withdrawn | Study of ALXN1830 Administered Subcutaneously in Adults With Generalized Myasthenia Gravis Generalized Myasthenia Gravis | Phase 2 | 2022-01-01 |
| Terminated | Ravulizumab Versus Placebo in Adult Participants With Dermatomyositis Dermatomyositis | Phase 2 / Phase 3 | 2021-11-19 |
| Completed | Study of ALXN1850 in Participants With Hypophosphatasia (HPP) Hypophosphatasia | Phase 1 | 2021-09-28 |
| Terminated | Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease Wilson Disease | Phase 3 | 2021-09-13 |
| Terminated | A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration Geographic Atrophy | Phase 2 | 2021-08-23 |
| Withdrawn | Study in Adult and Pediatric Patients With HSCT-TMA Stem Cell Transplant Complications, Thrombotic Microangiopathies | — | 2021-07-12 |
| Completed | Study of ALXN2050 in Healthy Adult Participants of Japanese Descent Healthy | Phase 1 | 2021-07-09 |
| Completed | Study of ALXN2050 in Participants With Renal Impairment Renal Impairment, Healthy | Phase 1 | 2021-07-08 |
| Withdrawn | ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia Warm Autoimmune Hemolytic Anemia | Phase 2 | 2021-07-01 |
| Terminated | Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger Thrombotic Microangiopathy | Phase 3 | 2021-06-29 |
| Terminated | Trial of Andexanet in Patients Receiving an Oral FXa Inhibitor Who Require Urgent Surgery Surgery | Phase 2 | 2021-06-27 |
| Completed | Drug Interaction Study of ALXN2050 With Fluconazole and Rifampin in Healthy Adult Participants Healthy | Phase 1 | 2021-06-23 |
| Completed | Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants Healthy | Phase 1 | 2021-05-30 |
| Completed | Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Ec Paroxysmal Nocturnal Hemoglobinuria | Phase 4 | 2021-04-14 |
| Completed | Study of Radiolabeled ALXN2050 in Healthy Adult Males Healthy | Phase 1 | 2021-04-12 |
| Terminated | A Study of ALXN1830 in Healthy Adult Participants Healthy | Phase 1 | 2021-03-17 |
| Completed | A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome Guillain-Barre Syndrome | Phase 3 | 2021-03-08 |
| Completed | Bioavailability Study of 2 Oral Formulations of ALXN1840 Healthy | Phase 1 | 2021-01-31 |
| Terminated | Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN) Lupus Nephritis, Immunoglobulin A Nephropathy | Phase 2 | 2021-01-19 |
| Completed | Study of ALXN1820 in Healthy Adult Participants Healthy | Phase 1 | 2021-01-13 |
| Completed | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Ha Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | 2020-12-16 |
| Completed | A Study of the Cardiac Effects of ALXN2050 in Healthy Adults Healthy | Phase 1 | 2020-12-12 |
| Completed | Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant Thrombotic Microangiopathy | Phase 3 | 2020-12-10 |
| Completed | Study of Ravulizumab in Pediatric Participants With HSCT-TMA Thrombotic Microangiopathy | Phase 3 | 2020-12-07 |
| Completed | Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated Wit Wilson Disease | Phase 2 | 2020-12-02 |
| Completed | A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM Symptomatic Transthyretin Amyloid Cardiomyopathy | Phase 3 | 2020-11-13 |
| Active Not Recruiting | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES AL Amyloidosis | Phase 3 | 2020-11-03 |
| Completed | Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants Wilson Disease | Phase 1 | 2020-10-21 |
| Completed | Study of the Metabolism of Danicopan in Healthy Adults Healthy | Phase 1 | 2020-10-20 |
| Completed | Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840 Wilson Disease | Phase 2 | 2020-09-07 |
| Completed | AZP-3601 SAD and MAD Study in Healthy Subjects and Patients With Hypoparathyroidism Chronic Hypoparathyroidism | Phase 1 / Phase 2 | 2020-09-07 |
| Active Not Recruiting | A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES AL Amyloidosis | Phase 3 | 2020-08-25 |
| Completed | Study of Danicopan in Participants of Japanese Descent Healthy | Phase 1 | 2020-08-17 |
| Completed | A Study of the Cardiac Effects of ALXN1840 in Healthy Adults Healthy | Phase 1 | 2020-07-24 |
| Completed | Phase 1 Study of ALXN1840 on the Metabolism of a CYP2C9 Substrate in Healthy Participants. Wilson Disease | Phase 1 | 2020-07-07 |
| Completed | Copper Balance in Healthy Participants Administered ALXN1840 Healthy | Phase 1 | 2020-07-01 |
| Completed | A Study to Assess ACH-0145228 When Administered as Immediate Release Tablet Versus Powder-In-Capsule in Health Healthy | Phase 1 | 2020-06-26 |
| Terminated | Efficacy and Safety Study of IV Ravulizumab in Patients With COVID-19 Severe Pneumonia COVID-19 Severe Pneumonia, Acute Lung Injury, Acute Respiratory Distress Syndrome | Phase 3 | 2020-05-10 |
| Withdrawn | Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease Hypophosphatasia | Phase 4 | 2020-04-01 |
| Terminated | An Efficacy and Safety Study of Ravulizumab in ALS Participants Amyotrophic Lateral Sclerosis, ALS | Phase 3 | 2020-03-30 |
| Completed | A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis AL Amyloidosis | Phase 2 | 2020-03-18 |
| Completed | A Study of the Effects of Food and Age on Danicopan Healthy | Phase 1 | 2020-02-21 |
| Terminated | A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder | Phase 2 / Phase 3 | 2020-01-14 |
| Completed | A Drug Interaction Study of ACH-0145228 Healthy | Phase 1 | 2019-12-22 |
| Terminated | Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | 2019-12-16 |
| Completed | An Efficacy and Safety Study of Ravulizumab in Adult Participants With NMOSD Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder | Phase 3 | 2019-12-09 |
| Recruiting | Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5IT Generalized Myasthenia Gravis | — | 2019-12-02 |
| Terminated | A Study of Single and Multiple SC Doses of ALXN1830 in Healthy Adult Participants Healthy | Phase 1 | 2019-11-12 |
| Completed | Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alf Hypophosphatasia | — | 2019-11-06 |
| Completed | Study of Subcutaneous and Intravenous ALXN1720 With and Without rHuPH20 in Healthy Subjects Healthy | Phase 1 | 2019-09-04 |
| Completed | A Drug Interaction Study of Danicopan Healthy | Phase 1 | 2019-07-28 |
| Completed | Study of Oral ALXN1840 at 2 Dose Strengths in Healthy Adults Healthy | Phase 1 | 2019-07-04 |
| Completed | Trial of Andexanet Alfa in ICrH Patients Receiving an Oral FXa Inhibitor Acute Intracranial Hemorrhage | Phase 4 | 2019-06-06 |
| Completed | Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis Generalized Myasthenia Gravis | Phase 3 | 2019-03-12 |
| Completed | Pharmacokinetic Study of Oral ALXN1840 in Japanese and Non-Japanese Adult Healthy Participants Wilson Disease | Phase 1 | 2019-02-20 |
| Completed | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglob Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | 2019-02-19 |
| Completed | A Study of Multiple Doses of ALXN2050 in Healthy Adults Healthy | Phase 1 | 2019-01-07 |
| Terminated | US Selumetinib Registry Neurofibromatosis Type 1, Plexiform Neurofibromas | — | 2019-01-04 |
| Completed | A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Grav Myasthenia Gravis, Myasthenia Gravis, Juvenile Form, Myasthenia Gravis, Generalized | Phase 3 | 2018-12-28 |
| Completed | Pharmacokinetic Study of Ravulizumab Administered Subcutaneously With Recombinant Human Hyaluronidase PH20 (rH Healthy | Phase 1 | 2018-08-09 |
| Completed | A Study of the Drug Interactions Between Danicopan and Cyclosporine, Tacrolimus, Antacids, and Omeprazole in H Healthy | Phase 1 | 2018-07-27 |
| Completed | A Study of the Cardiac Effects of Danicopan in Healthy Adults Healthy | Phase 1 | 2018-07-26 |
| Terminated | Study Evaluating Betrixaban in Pediatric Participants VTE Prophylaxis | Phase 1 | 2018-07-13 |
| Terminated | A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471 C3 Glomerulonephritis, C3 Glomerulopathy, Immune Complex Membranoproliferative Glomerulonephritis | Phase 2 | 2018-06-20 |
| Completed | A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G) C3 Glomerulopathy, C3 Glomerulonephritis, Dense Deposit Disease | Phase 2 | 2018-06-12 |
| Completed | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculiz Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | 2018-05-08 |
| Completed | Study of Danicopan in Participants With Hepatic Impairment Hepatic Impairment | Phase 1 | 2018-05-01 |
| Terminated | Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Wilson Disease | Phase 3 | 2018-02-22 |
| Completed | A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | 2018-02-22 |
| Completed | Study of Danicopan in Participants With Normal Kidney Function and Participants With Kidney Dysfunction Healthy, Renal Dysfunction | Phase 1 | 2018-01-24 |
| Terminated | A Safety Study of SYNT001 in Participants With Warm Autoimmune Hemolytic Anemia (WAIHA) Warm Autoimmune Hemolytic Anemia | Phase 1 / Phase 2 | 2018-01-10 |
| Completed | A Study of Modified Release Formulations of Danicopan in Healthy Adult Participants Healthy | Phase 1 | 2017-12-01 |
| Completed | A Study of Single-dose ALXN2050 in Healthy Adults Healthy | Phase 1 | 2017-11-28 |
| Completed | Study of Radiolabeled Danicopan in Healthy Male Participants Healthy | Phase 1 | 2017-09-14 |
| Completed | Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS) Atypical Hemolytic Uremic Syndrome (aHUS) | Phase 3 | 2017-08-31 |
| Completed | A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MP C3 Glomerulonephritis, Dense Deposit Disease, C3 Glomerulopathy | Phase 2 | 2017-08-09 |
| Terminated | A Safety and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus) Pemphigus, Pemphigus Vulgaris, Pemphigus Foliaceus | Phase 1 / Phase 2 | 2017-07-18 |
| Completed | A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | 2017-06-22 |
| Completed | ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Trea Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | 2017-05-17 |
| Completed | A Drug Interaction Study Between Danicopan and Midazolam, Fexofenadine, and Mycophenolate Mofetil in Healthy P Healthy | Phase 1 | 2017-04-18 |
| Completed | Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 2 | 2017-03-31 |
| Completed | Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemo Atypical Hemolytic Uremic Syndrome (aHUS) | Phase 3 | 2017-01-11 |
| Completed | ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Parox Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | 2016-12-12 |
| Terminated | Study of Samalizumab in Patients With Advanced Cancer Advanced Solid Tumors | Phase 1 | 2016-11-17 |
| Terminated | Screening for Lysosomal Acid Lipase Deficiency Lysosomal Acid Lipase Deficiency | — | 2016-11-01 |
| Completed | A Study of a Single Subcutaneous Dose of ALXN1210 in Healthy Adult Participants Healthy | Phase 1 | 2016-08-19 |
| Completed | Study of Different Forms of Danicopan (Tablet, Softgel Capsule, Liquid-filled Capsule) in Healthy Participants Healthy | Phase 1 | 2016-08-02 |
| Completed | Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatas Hypophosphatasia | Phase 2 | 2016-06-06 |
| Completed | Study of Multiple Doses of Danicopan in Healthy Participants Healthy | Phase 1 | 2016-05-21 |
| Completed | The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment Wilson Disease | — | 2016-05-19 |
| Completed | A Study of Single and Multiple Doses of ALXN1210 in Healthy, Adult Japanese Participants Healthy | Phase 1 | 2016-04-18 |
| Completed | Evaluation of Safety, Efficacy and Pharmacodynamic Effect of Bertilimumab in Patients With Bullous Pemphigoid Pemphigoid, Bullous | Phase 2 | 2016-02-29 |
| Completed | Study of a Single Dose of Danicopan in Healthy Participants Healthy | Phase 1 | 2016-02-04 |
| Completed | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 2 | 2016-01-04 |
| Completed | Dose-Escalation Study of ALXN1210 IV in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) PNH | Phase 1 / Phase 2 | 2015-11-12 |
| Terminated | Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndr Atypical Hemolytic Uremic Syndrome | — | 2015-11-04 |
| Terminated | A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B | Phase 1 / Phase 2 | 2015-10-15 |
| Terminated | Evaluation of Safety, Efficacy, Pharmacokinetic and Pharmacodynamic of Bertilimumab in Patients With Active Mo Ulcerative Colitis, Active Moderate, Ulcerative Colitis, Active Severe | Phase 2 | 2015-07-31 |
| Completed | A Study of Multiple Doses of ALXN1210 in Healthy Adult Participants Healthy | Phase 1 | 2015-07-28 |
| Completed | Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan Hypophosphatasia | Phase 4 | 2015-06-01 |
| Completed | A Study in Participants With Acute Major Bleeding to Evaluate the Ability of Andexanet Alfa to Reverse the Ant Bleeding | Phase 3 | 2015-04-10 |
| Completed | Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS I Mucopolysaccharidosis IIIB | Phase 1 / Phase 2 | 2015-01-22 |
| Enrolling By Invitation | Registry of Patients With Hypophosphatasia Hypophosphatasia (HPP) | — | 2015-01-20 |
| Completed | An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder | Phase 3 | 2015-01-12 |
| Terminated | A Study to Identify and Characterize LAL-D Patients in High-risk Populations Lysosomal Acid Lipase Deficiency | — | 2014-12-01 |
| Completed | Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients Wilson Disease | Phase 2 | 2014-11-24 |
| Terminated | A Phase 2A Study of ALXN1007 in Participants With Newly Diagnosed Acute Lower Gastrointestinal Graft-Versus-Ho Acute Graft-Versus-Host Disease, GIGVHD | Phase 2 | 2014-11-14 |
| Completed | Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthen Refractory Generalized Myasthenia Gravis | Phase 3 | 2014-11-12 |
| Completed | Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients With AL Amyloidosis AL Amyloidosis | Phase 1 | 2014-10-30 |
| Terminated | Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharid MPS IIIB (Sanfilippo B Syndrome) | — | 2014-09-14 |
| Completed | A Study of a Single Dose of ALXN1210 in Healthy Participants Healthy | Phase 1 | 2014-08-27 |
| Completed | Prevention of Delayed Graft Function Using Eculizumab Therapy (PROTECT Study) Delayed Graft Function | Phase 2 / Phase 3 | 2014-08-21 |
| Completed | Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who S Hypophosphatasia, Bone Disease | — | 2014-08-01 |
| Completed | Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency Lysosomal Acid Lipase Deficiency | Phase 2 | 2014-06-24 |
| Terminated | Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Lysosomal Acid Lipase Deficiency | Phase 2 | 2014-06-06 |
| Terminated | A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study) Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder | Phase 3 | 2014-04-11 |
| Completed | A Study of ALXN1840 (Coated and Non-coated) Administered With And Without Omeprazole In Healthy Adults Healthy | Phase 1 | 2014-04-07 |
| Terminated | Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome Antiphospholipid (aPL)-Positive | Phase 2 | 2014-04-01 |
| Completed | Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP) Hypophosphatasia (HPP) | — | 2014-03-01 |
| Completed | A Study of ALXN1840 (Non-coated) Administered With And Without Omeprazole In Healthy Adults Healthy | Phase 1 | 2014-01-20 |
| Completed | Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal MPS IIIB (Sanfilippo B Syndrome) | — | 2013-12-01 |
| Completed | Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study) Refractory Generalized Myasthenia Gravis | Phase 3 | 2013-12-01 |
| Completed | Phase 1/2a Dose Escalation Study in Participants With CLL, SLL, or NHL Follicular Lymphoma (FL/Indolent NHL), Aggressive NHL (a NHL), Chronic Lymphocytic Leukemia (CLL) / Small Lymphocytic Lymphoma (SLL) | Phase 1 / Phase 2 | 2013-08-30 |
| Completed | A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB MPS IIIB (Sanfilippo Syndrome) | — | 2013-07-01 |
| Completed | Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALXN1007 in Healthy Male Healthy Subjects | Phase 1 | 2013-06-01 |
| Recruiting | Lysosomal Acid Lipase (LAL) Deficiency Registry Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease | — | 2013-05-30 |
| Completed | Safety, Tolerability, and Efficacy of 12-weeks of Sovaprevir, ACH-3102, and Ribavirin in Treatment-naive GT-1 Hepatitis C, Chronic | Phase 2 | 2013-04-01 |
| Recruiting | Atypical Hemolytic-Uremic Syndrome (aHUS) Registry Atypical Hemolytic-Uremic Syndrome | — | 2013-03-18 |
| Completed | Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase D Lysosomal Acid Lipase Deficiency | Phase 3 | 2013-01-22 |
| Completed | Evaluation of Safety, Tolerability, and Antiviral Activity of ACH-0143102 Plus Ribavirin In Treatment-naive He Chronic Hepatitis C Infection | Phase 1 | 2012-09-01 |
| Completed | Safety & Efficacy Of Eculizumab In The Prevention Of AMR In Sensitized Recipients Of A Kidney Transplant From Stage V Chronic Kidney Disease | Phase 2 | 2012-08-29 |
| Completed | A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia Hypophosphatasia (HPP) | — | 2012-08-01 |
| Completed | The Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS) Atypical Hemolytic Uremic Syndrome (aHUS) | Phase 2 | 2012-05-16 |
| Completed | To Characterize the Safety and Tolerability of Eculizumab in Two Japanese aHUS Patients ATYPICAL HEMOLYTIC UREMIC SYNDROME (AHUS) | — | 2012-05-01 |
| Terminated | aHUS Observational Long Term Follow-Up Atypical Hemolytic Uremic Syndrome | — | 2012-03-01 |
| Completed | Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study Paroxysmal Nocturnal Hemoglobinuria, PNH | — | 2012-03-01 |
| Completed | Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in A Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency | Phase 2 | 2011-12-12 |
| Terminated | Safety & Efficacy of Eculizumab to Prevent AMR in Living Donor Kidney Transplant Recipients Requiring Desensit Antibody Mediated Rejection | Phase 2 | 2011-11-02 |
| Completed | Study to Evaluate Safety, Tolerability, PK and PD of ALXN1007 in Healthy Subjects Healthy | Phase 1 | 2011-11-01 |
| Completed | Safety and Efficacy Study of Eculizumab in Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (S Shiga-like Toxin-producing Escherichia Coli | Phase 2 / Phase 3 | 2011-07-01 |
| Completed | An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phe Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency | — | 2011-06-01 |
| Completed | Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Gro Lysosomal Acid Lipase Deficiency, Wolman Disease | Phase 2 / Phase 3 | 2011-05-04 |
| Completed | Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Ac Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency | Phase 1 / Phase 2 | 2011-04-25 |
| Terminated | Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 1 | 2011-04-01 |
| Completed | A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype Lysosomal Acid Lipase Deficiency, Wolman Disease | — | 2010-11-01 |
| Completed | Safety, Tolerability, and Antiviral Activity of ACH-0141625 or Placebo in Combination With Peginterferon and R Hepatitis C | Phase 2 | 2010-09-01 |
| Completed | An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Atypical Hemolytic-Uremic Syndrome | Phase 2 | 2010-09-01 |
| Completed | Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP) Hypophosphatasia | Phase 2 / Phase 3 | 2010-07-01 |
| Completed | An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Synd Atypical Hemolytic-Uremic Syndrome | Phase 2 | 2010-07-01 |
| Completed | Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP) Hypophosphatasia | Phase 2 | 2010-06-01 |
| Completed | Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) Hypophosphatasia (HPP) | Phase 2 | 2010-04-01 |
| Completed | Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria Hemoglobinuria, Paroxysmal | Phase 4 | 2009-10-02 |
| Completed | Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP) Hypophosphatasia (HPP) | Phase 2 | 2009-09-01 |
| Completed | A Retrospective, Observational, Non-interventional Trial to Assess Eculizumab Treatment Effect in Patients Wit Atypical Hemolytic Uremic Syndrome (aHUS) | — | 2009-08-31 |
| Completed | Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic U Atypical Hemolytic Uremic Syndrome | Phase 2 | 2009-07-01 |
| Completed | Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS Atypical Hemolytic Uremic Syndrome | Phase 2 | 2009-07-01 |
| Withdrawn | Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP) Hypophosphatasia | Phase 2 | 2009-07-01 |
| Completed | Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS Atypical Hemolytic Uremic Syndrome | Phase 2 | 2009-05-01 |
| Completed | Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS Atypical Hemolytic Uremic Syndrome | Phase 2 | 2009-05-01 |
| Completed | Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophospha Hypophosphatasia | Phase 2 | 2009-04-01 |
| Terminated | Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis Myasthenia Gravis | Phase 2 | 2008-10-01 |
| Completed | Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) Hypophosphatasia (HPP) | Phase 1 / Phase 2 | 2008-09-01 |
| Completed | Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adu Hypophosphatasia (HPP) | Phase 1 | 2008-08-01 |
| Terminated | Safety and Dose Ranging Study of Samalizumab to Treat Relapsing or Refractory CLL or MM B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma | Phase 1 / Phase 2 | 2008-06-19 |
| Completed | An Open-Label Treatment Protocol to Provide Continued Elvucitabine Treatment HIV Infections | Phase 2 | 2008-05-01 |
| Completed | E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | 2008-04-01 |
| Completed | Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Paroxysmal Nocturnal Hemoglobinuria | Phase 2 | 2007-11-01 |
| Completed | Safety and Efficacy Study of Eculizumab in Patients With Mild Allergic Asthma Allergic Asthma | Phase 2 | 2007-09-01 |
| Terminated | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Antiviral Activity of ACH-0137171 in Pa HCV Infection | Phase 2 | 2006-11-30 |
| Completed | Open-Label, 48-Week Extension Study of Elvucitabine in Combination With Background Antiretroviral Therapy (ART Human Immunodeficiency Virus (HIV)-1 Infection | Phase 2 | 2006-09-01 |
| Completed | Elvucitabine/Efavirenz/Tenofovir Versus Lamivudine/Efavirenz/Tenofovir in Human Immunodeficiency Virus (HIV)-1 HIV Infections | Phase 2 | 2006-05-01 |
| Completed | Study of Once Daily Elvucitabine Versus Lamivudine in Participants With a Documented M184V Mutation HIV Infections | Phase 1 / Phase 2 | 2006-03-31 |
| Completed | Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Paroxysmal Hemoglobinuria, Nocturnal | Phase 3 | 2005-05-01 |
| Completed | Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab Hemoglobinuria, Paroxysmal | Phase 3 | 2004-12-01 |
| Completed | Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry Paroxysmal Nocturnal Hemoglobinuria | — | 2004-10-29 |
| Completed | Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Hemoglobinuria, Paroxysmal | Phase 3 | 2004-10-01 |
| Completed | Pexelizumab in Patients Undergoing Coronary Artery Bypass Grafting With Cardiopulmonary Bypass (PRIMO-CABG II) Coronary Artery Disease | Phase 3 | 2004-07-01 |
| Terminated | Safety and Antiviral Study of ACH126, 433 (b-L-Fd4C) in Adults With Lamivudine-resistant Chronic Hepatitis B Chronic Hepatitis B | Phase 2 | 2002-07-01 |
| Completed | Safety and Antiviral Activity Study of ACH-126,443 (Beta-L-Fd4C) in Treatment-naive Adults With Chronic Hepati Hepatitis B, Chronic | Phase 2 | 2002-02-01 |
| Completed | Effect of Pexelizumab on All-Cause Mortality and Myocardial Infarction in Patients Undergoing Coronary Artery Cardio-pulmonary Bypass, Coronary Artery Bypass Graft | Phase 3 | 2002-01-01 |
| Terminated | Safety and Antiviral Study of ACH-126, 443 (Beta-L-Fd4C) in the Treatment of Adults With Chronic Hepatitis B I Chronic Hepatitis B | N/A | — |
| No Longer Available | Expanded Access Program for Participants Who Completed Study 13-601 and Continue to Clinically Benefit From Ce Relapsed/Refractory Chronic Lymphocytic Leukemia, Non-hodgkin's Lymphoma | — | — |
| Approved For Marketing | Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophospha Hypophosphatasia | — | — |
| Terminated | Safety and Antiviral Study of ACH-126, 443 (Beta-L-Fd4C) in the Treatment of Adults With HIV Infection and Mod HIV Infections | Phase 2 | — |
| No Longer Available | Early Access Program for ALXN1840 in Patients With Wilson Disease Wilson Disease | — | — |
| Approved For Marketing | The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol Hemoglobinuria, Paroxysmal | — | — |
| Available | Danicopan Early Access Program Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis | — | — |
| No Longer Available | COVID-19 Soliris Expanded Access Protocol Covid19 | — | — |
| Completed | Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure S Hemoglobinuria | — | — |
| No Longer Available | An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency Lysosomal Acid Lipase Deficiency | — | — |
| No Longer Available | SOLIRIS® (Eculizumab) Treatment of Participants With COVID-19 COVID-19, Pneumonia, Viral, Acute Lung Injury/Acute Respiratory Distress Syndrome (ARDS) | — | — |