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Alexion Pharmaceuticals clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine and the EU Clinical Trials Information System (CTIS) — current trials in our USA, UK, China, Ireland and EU (CTIS) registers with Alexion Pharmaceuticals as lead sponsor. Synced 26 Sep 2026
41Current trials
18Recruiting now
14Drugs in these trials
5Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

Alexion Pharmaceuticals is the lead sponsor of 41 current clinical trials in our USA, UK, China, Ireland and EU (CTIS) registers. 18 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are Ravulizumab, Danicopan and ALXN1850. The most-studied condition is Generalized Myasthenia Gravis.

Company profile on this site (news, sites, medicines, approvals): AstraZeneca (Alexion) (GB) →

ClinicalTrials.gov trials by status

  • Recruiting1846%
  • Not yet open13%
  • Active, closed1949%
  • By invitation13%

Trials by phase

  • Phase 31949%
  • Observational1128%
  • Phase 238%
  • Phase 125%
  • Phase 1/225%
  • Phase 413%
  • Phase 2/313%
01

Clinical trials sponsored by Alexion Pharmaceuticals

39 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
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 · Delayed Graft Function, DGF, Kidney Transplant
Phase 3 Recruiting 2025-05 UK, USA, China
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
NCT07081646 · Relapsed AL Amyloidosis, Refractory AL Amyloidosis, Light Chain Amyloidosis
Drug: AZD0120
Phase 1/2 Recruiting 2025-08 UK, USA
Lysosomal Acid Lipase (LAL) Deficiency Registry
NCT01633489 · ClinicalTrials.gov ↗ · Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease
Observational Recruiting 2013-05 Ireland, UK, USA
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
NCT06449001 · ClinicalTrials.gov ↗ · Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
Drug: Danicopan
Phase 3 Recruiting 2025-08 UK
Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants
NCT07352423 · ClinicalTrials.gov ↗ · Healthy Adult Participants
Phase 1 Recruiting 2026-03 UK
Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)
NCT07157787 · ClinicalTrials.gov ↗ · Primary Membranous Nephropathy
Phase 2 Recruiting 2025-09 UK, USA, China
Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis
NCT07160608 · ClinicalTrials.gov ↗ · Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Phase 2 Recruiting 2025-11 UK, China
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
NCT01522183 · ClinicalTrials.gov ↗ · Atypical Hemolytic-Uremic Syndrome
Observational Recruiting 2013-03 UK, USA
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
NCT07218887 · ClinicalTrials.gov ↗ · BAG3 Mutation Associated Dilated Cardiomyopathy
Phase 1/2 Recruiting 2025-10 UK, USA
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
NCT06312644 · ClinicalTrials.gov ↗ · Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH)
Observational Recruiting 2024-12 UK, USA
Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)
NCT04202341 · ClinicalTrials.gov ↗ · Generalized Myasthenia Gravis
Observational Recruiting 2019-12 USA, China
Study of Ravulizumab in Pediatric Participants With Primary IgAN
NCT07024563 · ClinicalTrials.gov ↗ · IgAN, IgAVN, Immunoglobulin A Nephropathy
Phase 3 Recruiting 2025-06 USA, China
A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
NCT07221838 · ClinicalTrials.gov ↗ · Generalized Myasthenia Gravis, gMG
Phase 4 Recruiting 2026-03 USA
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
NCT05966467 · ClinicalTrials.gov ↗ · Neuromyelitis Optica Spectrum Disorder, NMOSD, AQP4+ NMOSD
Observational Recruiting 2024-02 USA, China
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
NCT06607627 · ClinicalTrials.gov ↗ · Generalized Myasthenia Gravis, gMG
Phase 3 Recruiting 2024-11 USA
A Prospective Sub-Study of the Global Hypophosphatasia Registry
NCT05234567 · ClinicalTrials.gov ↗ · Hypophosphatasia
Observational Recruiting 2022-08 USA
Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)
NCT07596784 · ClinicalTrials.gov ↗ · Generalized Myasthenia Gravis, gMG
Phase 3 Recruiting 2026-07 China
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)
NCT07557420 · ClinicalTrials.gov ↗ · NMOSD, Neuromyelitis Optica Spectrum Disorder
Phase 3 Recruiting 2026-07 China
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
NCT07789483 · Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
Drug: ALXN2220
Phase 3 Not yet open 2027-11 Ireland, UK, USA, China
Registry of Patients With Hypophosphatasia
NCT02306720 · Hypophosphatasia (HPP)
Observational By invitation 2015-01 UK, USA
Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN)
NCT06291376 · Immunoglobulin A Nephropathy, IgAN
Phase 3 Active, closed 2024-03 UK, USA, China
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
NCT05556096 · Generalized Myasthenia Gravis
Phase 3 Active, closed 2022-11 UK, USA, China
Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation
NCT06744647 · Antibody-Mediated Rejection, Kidney Transplantation, Biopsy-proven Histologic Scores
Drug: ALXN2030
Phase 2 Active, closed 2025-03 UK, USA, China
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
NCT06183931 · ClinicalTrials.gov ↗ · Transthyretin Amyloid Cardiomyopathy
Drug: ALXN2220
Phase 3 Active, closed 2024-01 Ireland, UK, USA, China
Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants
NCT05501717 · Healthy
Drug: ALXN2030
Phase 1 Active, closed 2022-08 UK
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
NCT06079281 · Hypophosphatasia
Drug: ALXN1850
Phase 3 Active, closed 2024-01 UK, USA, China
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
NCT07413679 · Paroxysmal Nocturnal Hemoglobinuria, PNH
Drug: Danicopan
Observational Active, closed 2025-08 USA
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
NCT04512235 · ClinicalTrials.gov ↗ · AL Amyloidosis
Phase 3 Active, closed 2020-11 UK, USA, China
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
NCT05981092 · ClinicalTrials.gov ↗ · Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
Observational Active, closed 2022-10 UK, USA
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
NCT06079359 · ClinicalTrials.gov ↗ · Hypophosphatasia
Drug: ALXN1850
Phase 3 Active, closed 2024-05 UK, USA, China
Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism
NCT05778071 · ClinicalTrials.gov ↗ · Chronic Hypoparathyroidism, Endocrine System Diseases, Parathyroid Diseases
Phase 3 Active, closed 2023-06 UK, USA
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
NCT06079372 · ClinicalTrials.gov ↗ · Hypophosphatasia
Phase 3 Active, closed 2024-04 UK, USA
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 · ClinicalTrials.gov ↗ · Paroxysmal Nocturnal Hemoglobinuria
Drug: Danicopan
Phase 3 Active, closed 2022-10 UK, USA
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)
NCT04504825 · ClinicalTrials.gov ↗ · AL Amyloidosis
Phase 3 Active, closed 2020-08 UK, USA, China
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
NCT05506254 · ClinicalTrials.gov ↗ · Methylmalonic Acidemia
Observational Active, closed 2022-07 USA
Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD
NCT06724809 · ClinicalTrials.gov ↗ · NMOSD, Neuromyelitis Optica Spectrum Disorders
Phase 3 Active, closed 2025-01 China
Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis
NCT05644561 · ClinicalTrials.gov ↗ · Generalized Myasthenia Gravis, gMG
Phase 3 Active, closed 2023-06 USA
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
NCT07413250 · ClinicalTrials.gov ↗ · Paroxysmal Nocturnal Hemoglobinuria, PNH
Drug: Danicopan
Observational Active, closed 2026-01 USA
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
NCT05346354 · ClinicalTrials.gov ↗ · Neuromyelitis Optica Spectrum Disorder
Phase 2/3 Active, closed 2022-06 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Alexion Pharmaceuticals in the EU trials register (CTIS)

2 trials

Authorised or ongoing trials in the EU Clinical Trials Information System with Alexion Pharmaceuticals as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

StudyPhaseStatusCountries
OCTAGON:A Phase 4, Single Arm Study to Investigate Oral Corticosteroid Tapering in Adult Patients with Generalized Myasthenia Gravis Treated with Ravulizumab
2025-522555-26-00 · CTIS ↗ · Nervous System Diseases
Phase 4 Authorised Italy, Germany
A Phase 1/2, Open-Label, Dose Finding Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants with Symptomatic BAG3 Mutation-Associated Dilated Cardiomyopathy
2024-519674-40-00 · CTIS ↗ · Cardiovascular Diseases
Drug: Sirolimus
Phase 1 Ongoing Spain, Italy, Germany
03

Drugs in Alexion Pharmaceuticals trials

14 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

04

Conditions in Alexion Pharmaceuticals trials

49 conditions
Generalized Myasthenia Gravis6Hypophosphatasia4Paroxysmal Nocturnal Hemoglobinuria4gMG4PNH3Neuromyelitis Optica Spectrum Disorder3NMOSD3Immunoglobulin A Nephropathy2IgAN2AL Amyloidosis2Transthyretin Amyloid Cardiomyopathy (ATTR-CM)1Transthyretin Amyloid Cardiomyopathy1Lysosomal Acid Lipase Deficiency1Cholesterol Ester Storage Disease1Wolman Disease1Acid Cholesteryl Ester Hydrolase Deficiency, Type 21Antibody-Mediated Rejection1Kidney Transplantation1Biopsy-proven Histologic Scores1AMR1Delayed Graft Function1DGF1Kidney Transplant1Hypophosphatasia (HPP)1
05

By trial register

RegisterTrialsRecruiting
Clinical trials in USA3213
Clinical trials in UK2510
Clinical trials in China188
Clinical trials in Ireland31
EU register (CTIS)2—

A trial with sites in more than one country appears in each of those registers.

06

Other sponsors in Generalized Myasthenia Gravis

SponsorTrials
argenx5
UCB Biopharma2
Kyverna Therapeutics1
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany1
Candid Therapeutics1

Sponsors with the most current trials registered for this condition.

07

Questions about Alexion Pharmaceuticals trials

How many clinical trials does Alexion Pharmaceuticals sponsor?

Alexion Pharmaceuticals is the lead sponsor of 39 current trials on ClinicalTrials.gov in our USA, UK, China and Ireland registers, and 2 authorised or ongoing trials in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Alexion Pharmaceuticals trials recruiting?

Yes. 18 of its 39 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Alexion Pharmaceuticals testing?

Its current trials name 14 drugs and biologicals as interventions. The most frequent are Ravulizumab (9), Danicopan (4), ALXN1850 (3), ALXN2030 (2) and ALXN2220 (2). The number in brackets is how many of its trials name each drug.

Does Alexion Pharmaceuticals have clinical trials in Ireland?

Yes. 3 trials in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.