Alexion Pharmaceuticals
Alexion Pharmaceuticals sponsors 162 registered US clinical trials on ClinicalTrials.gov, 17 of them currently recruiting, and 93 completed. 45 of these trials are in Phase 3-4 (later-stage) and 90 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Paroxysmal Nocturnal Hemoglobinuria, with 5 trials.
Trial Pipeline
162 total, page 3 of 4
An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
NCT02003144
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
NCT02345421
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
NCT02273596
A Phase 2A Study of ALXN1007 in Participants With Newly Diagnosed Acute Lower Gastrointestinal Graft-Versus-Host Disease
NCT02245412
Extension Study of ECU-MG-301 to Evaluate Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis
NCT02301624
Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients With AL Amyloidosis
NCT02245867
Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB
NCT02293408
Prevention of Delayed Graft Function Using Eculizumab Therapy (PROTECT Study)
NCT02145182
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
NCT02235493
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
NCT02112994
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
NCT02193867
A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
NCT01892345
A Study of ALXN1840 (Coated and Non-coated) Administered With And Without Omeprazole In Healthy Adults
NCT05319912
Phase IIa Trial of ALXN1007 for the Treatment of Non-criteria Manifestations of Antiphospholipid Syndrome
NCT02128269
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
NCT02104219
A Study of ALXN1840 (Non-coated) Administered With And Without Omeprazole In Healthy Adults
NCT05319899
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
NCT01997229
Phase 1/2a Dose Escalation Study in Participants With CLL, SLL, or NHL
NCT01994382
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
NCT02293382
Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ALXN1007 in Healthy Male and Female Subjects
NCT01883544
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
NCT01757184
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
NCT01419028
Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study
NCT01412047
aHUS Observational Long Term Follow-Up
NCT01522170
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
NCT01488097
Safety & Efficacy of Eculizumab to Prevent AMR in Living Donor Kidney Transplant Recipients Requiring Desensitization
NCT01399593
Study to Evaluate Safety, Tolerability, PK and PD of ALXN1007 in Healthy Subjects
NCT01454986
An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
NCT01528917
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
NCT01371825
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
NCT01307098
Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT01335165
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
NCT01358370
An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
NCT01193348
Safety, Tolerability, and Antiviral Activity of ACH-0141625 or Placebo in Combination With Peginterferon and Ribavirin in Hepatitis C Virus (HCV) Positive Participants
NCT01180790
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
NCT01176266
An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome
NCT01194973
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
NCT01163149
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
NCT01203826
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT00867932
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
NCT00952484
A Retrospective, Observational, Non-interventional Trial to Assess Eculizumab Treatment Effect in Patients With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT01770951
Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS
NCT00844545
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS
NCT00844844
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
NCT01205152
Safety and Efficacy Study of Eculizumab in Patients With Refractory Generalized Myasthenia Gravis
NCT00727194
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
NCT00744042
Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
NCT00739505
Safety and Dose Ranging Study of Samalizumab to Treat Relapsing or Refractory CLL or MM
NCT00648739
An Open-Label Treatment Protocol to Provide Continued Elvucitabine Treatment
NCT00675844
Safety and Efficacy Study of Adjunctive Antiplatelet Therapy Prior to Primary PCI in Patients With STEMI
NCT00546260
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 43 |
| Phase 2 | 47 |
| Phase 3 | 42 |
| Phase 4 | 3 |
What the Pipeline for Alexion Pharmaceuticals Shows
According to the ClinicalTrials.gov registry, Alexion Pharmaceuticals is linked to 162 US clinical trials across every stage of research activity. Of those, 17 studies are currently recruiting, about 10% of the sponsor's indexed portfolio, and 93 are already marked complete, representing roughly 57% of the total.
The phase mix for Alexion Pharmaceuticals reports 45 late-stage studies (Phase 3 and Phase 4 combined) and 90 earlier-phase studies (Phase 1 and Phase 2).
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.
How to read these pipeline numbers
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.
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.
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.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.