Alexion Pharmaceuticals

109 total trials 74 currently recruiting 19 completed

Trial Pipeline

RECRUITING Phase 2

ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly

NCT07037420

RECRUITING Phase 1

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

NCT07218887

RECRUITING Phase 2

Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)

NCT07157787

RECRUITING Phase 1

A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.

NCT07081646

RECRUITING Phase 3

Study of Ravulizumab in Pediatric Participants With Primary IgAN

NCT07024563

RECRUITING Phase 3

Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab Administered Intravenously in Adult Participants at High Risk of Delayed Graft Function After Kidney Transplantation

NCT06830798

RECRUITING Phase 2

Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation

NCT06744647

RECRUITING

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

NCT06312644

RECRUITING Phase 3

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis

NCT06607627

RECRUITING Phase 3

Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN)

NCT06291376

RECRUITING

Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies

NCT05966467

RECRUITING

A Prospective Sub-Study of the Global Hypophosphatasia Registry

NCT05234567

RECRUITING Phase 2

Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

NCT05346354

RECRUITING

Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)

NCT04202341

RECRUITING

Lysosomal Acid Lipase (LAL) Deficiency Registry

NCT01633489

RECRUITING

Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

NCT01522183

ACTIVE NOT RECRUITING

Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

NCT07413250

ACTIVE NOT RECRUITING

Long-term Safety of Danicopan: IPIG Registry-based Cohort Study

NCT07413679

ACTIVE NOT RECRUITING Phase 3

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP

NCT06079359

ACTIVE NOT RECRUITING Phase 3

Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa

NCT06079372

ACTIVE NOT RECRUITING Phase 3

Study of ALXN2220 Versus Placebo in Adults With ATTR-CM

NCT06183931

ACTIVE NOT RECRUITING Phase 3

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa

NCT06079281

ACTIVE NOT RECRUITING Phase 3

Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

NCT05644561

ACTIVE NOT RECRUITING Phase 3

Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism

NCT05778071

ACTIVE NOT RECRUITING Phase 3

ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE

NCT05746559

ACTIVE NOT RECRUITING Phase 3

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

NCT05556096

ACTIVE NOT RECRUITING Phase 3

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

NCT05389449

ACTIVE NOT RECRUITING

A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

NCT05981092

ACTIVE NOT RECRUITING

Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy

NCT05506254

ACTIVE NOT RECRUITING Phase 3

Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

NCT04543591

ACTIVE NOT RECRUITING Phase 3

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

NCT04512235

ACTIVE NOT RECRUITING Phase 3

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

NCT04504825

COMPLETED Phase 1

Drug-Drug Interaction (DDI) Study of ALXN2050 in Healthy Adult Participants

NCT05202145

COMPLETED Phase 1

A Study to Assess ACH-0145228 When Administered as Immediate Release Tablet Versus Powder-In-Capsule in Healthy Adult Participants

NCT04551586

COMPLETED Phase 3

Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

NCT03748823

COMPLETED Phase 3

A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria

NCT03406507

COMPLETED Phase 1

Study of Danicopan in Participants With Normal Kidney Function and Participants With Kidney Dysfunction

NCT04935294

COMPLETED Phase 3

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

NCT03056040

COMPLETED Phase 3

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT02946463

COMPLETED

The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment

NCT02763215

COMPLETED Phase 1

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049

COMPLETED Phase 2

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

NCT02112994

COMPLETED Phase 1

A Study of ALXN1840 (Non-coated) Administered With And Without Omeprazole In Healthy Adults

NCT05319899

COMPLETED Phase 3

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

NCT01757184

COMPLETED

A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)

NCT01419028

COMPLETED Phase 2

Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

NCT01205152

COMPLETED Phase 2

An Open-Label Treatment Protocol to Provide Continued Elvucitabine Treatment

NCT00675844

COMPLETED Phase 2

Elvucitabine/Efavirenz/Tenofovir Versus Lamivudine/Efavirenz/Tenofovir in Human Immunodeficiency Virus (HIV)-1 Infected, Treatment-naive Participants

NCT00350272

COMPLETED Phase 1

Study of Once Daily Elvucitabine Versus Lamivudine in Participants With a Documented M184V Mutation

NCT00312039

COMPLETED Phase 3

Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT00122317

COMPLETED Phase 3

Pexelizumab in Patients Undergoing Coronary Artery Bypass Grafting With Cardiopulmonary Bypass (PRIMO-CABG II)

NCT00088179

What the Pipeline for Alexion Pharmaceuticals Shows

According to the ClinicalTrials.gov registry, Alexion Pharmaceuticals is linked to 109 US clinical trials across every stage of research activity. Of those, 74 studies are currently recruiting — about 68% of the sponsor's indexed portfolio — and 19 are already marked complete, representing roughly 17% of the total. Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.

The phase mix for Alexion Pharmaceuticals reports 23 late-stage studies (Phase 3 and Phase 4 combined) and 16 earlier-phase studies (Phase 1 and Phase 2). A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work — safety, dosing, and early signal detection — and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.

The top therapeutic focus area indexed for Alexion Pharmaceuticals is Paroxysmal Nocturnal Hemoglobinuria with 5 linked trials, and 9 other condition areas appear in the top list above. That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice — verify current details directly with ClinicalTrials.gov before relying on any figure here.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial