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Alexion Pharmaceuticals Inc.
Sponsor

Alexion Pharmaceuticals Inc.

United States 121 Seaport Boulevard, 02210-2050, United States

About the sponsor

The sponsor demonstrates substantial commitment to advancing therapies for rare blood disorders and complement-mediated conditions. Research encompasses paroxysmal nocturnal hemoglobinuria, a life-threatening disorder characterized by hemolysis and thrombosis, with investigations into both intravascular and extravascular hemolytic activity. The portfolio extends to atypical hemolytic uremic syndrome, addressing complement dysregulation that leads to thrombotic microangiopathy.

  • Complement component 5 inhibition strategies
  • Treatment of clinically significant extravascular hemolysis
  • Hematopoietic stem cell transplant-associated thrombotic microangiopathy
  • Hemoglobin stabilization and transfusion independence

Therapeutic development includes both intravenous and subcutaneous formulations designed for pediatric and adult populations, with particular emphasis on pharmacokinetic and pharmacodynamic profiling across different age groups.

About Specializations

Alexion Pharmaceuticals Inc. runs clinical trials in conditions such as IgA nephropathy, Hypophosphatasia, Myasthenia gravis, Paroxysmal nocturnal haemoglobinuria, among others.

Recruiting now

Ongoing trials

Clinical trials run by this sponsor – those enrolling participants first.

Condition
Country
City
Type of trial
Recruitment status
Quick filters

30 clinical trials

Familial dilated cardiomyopathy

Recruiting

Study of ALXN2350 gene therapy in adults with BAG3 mutation-associated dilated cardiomyopathy: A safety and efficacy trial

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:AAV9-CTNT-COBAG3
  • Sponsor:Alexion Pharmaceuticals Inc.

Renal transplant+1

Recruiting

Study of Ravulizumab to Prevent Delayed Graft Function in Adult Patients After Kidney Transplant

Registered drug
Countries:AustriaAustria
  • Participants:18–64 years · 65+ years
  • Substances:Ravulizumab
  • Sponsor:Alexion Pharmaceuticals Inc.

IgA nephropathy

Recruiting

Study on Ravulizumab for Children with IgA Nephropathy (Ages 2-17)

Registered drugNo placebo
Countries:ItalyItaly
  • Participants:0–17 years
  • Substances:Ravulizumab
  • Sponsor:Alexion Pharmaceuticals Inc.

Myasthenia gravis

Recruiting

Study of Gefurulimab for Children and Teens with Generalized Myasthenia Gravis

InvestigationalNo placebo
Countries:PolandPoland
  • Participants:0–17 years
  • Substances:Gefurulimab
  • Sponsor:Alexion Pharmaceuticals Inc.

Paroxysmal nocturnal haemoglobinuria

Recruiting

Study of Danicopan with Ravulizumab or Eculizumab for Children with Paroxysmal Nocturnal Hemoglobinuria and Extravascular Hemolysis

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:Danicopan
  • Sponsor:Alexion Pharmaceuticals Inc.

Study on Ravulizumab for Children Aged 6 to 17 with Generalized Myasthenia Gravis

Recruiting
Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Ravulizumab
  • Sponsor:Alexion Pharmaceuticals Inc.

Myasthenia gravis

Not yet recruiting

Oral corticosteroid tapering with ravulizumab, prednisone and prednisolone in adult patients with generalized myasthenia gravis

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:18–64 years · 65+ years
  • Substances:Prednisolone
  • Sponsor:Alexion Pharmaceuticals Inc.

Hypophosphatasia

Not yet recruiting

Study of ALXN1850 for Children Aged 2 to 12 with Hypophosphatasia Who Have Not Previously Received Asfotase Alfa Treatment

Investigational
Countries:BelgiumBelgium
  • Participants:0–17 years
  • Substances:ALXN1850
  • Sponsor:Alexion Pharmaceuticals Inc.

Hypophosphatasia

Not yet recruiting

Study on ALXN1850 and Asfotase Alfa for Children Aged 2-12 with Hypophosphatasia Previously Treated with Asfotase Alfa

Registered drugNo placebo
Countries:FranceFrance
  • Participants:0–17 years
  • Substances:ALXN1850
  • Sponsor:Alexion Pharmaceuticals Inc.

Anti-neutrophil cytoplasmic antibody positive vasculitis

Not recruiting

A Study of Tarperprumig Safety and Effectiveness in Adults with Blood Vessel Inflammation Caused by ANCA Antibodies

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Sponsor:Alexion Pharmaceuticals Inc.

Nephropathy

Not recruiting

A Study of ALXN1920 Compared to Placebo in Adults with Primary Membranous Nephropathy at High Risk for Disease Progression

Investigational
Countries:FranceFrance
  • Participants:18–64 years · 65+ years
  • Substances:ALXN1920
  • Sponsor:Alexion Pharmaceuticals Inc.

Transplant rejection

Not recruiting

A study testing ALXN2030 for adults with antibody-mediated rejection after kidney transplantation

Investigational
Countries:SpainSpain
  • Participants:18–64 years · 65+ years
  • Substances:RNA, (AM-SP-UM-CM-AM-AM-CM-UM-(2'-DEOXY-2'-FLUORO)C-(2'-DEOXY-2'-FLUORO)A-(2'-DEOXY-2'-FLUORO)C-(2'-DEOXY-2'-FLUORO)C-UM-GM-UM-AM-AM-UM-AM-AM-AM-GM-CM-AM-GM-CM-CM-GM-[2'-O-[[2-[2-[[5-[[2-(ACETYLAMINO)-2-DEOXY-BETA-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]ETHOXY]ETHOXY]METHYL]]A-[2'-O-[[2-[2-[[5-[[2-(ACETYLAMINO)-2-DEOXY-BETA-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]ETHOXY]ETHOXY]METHYL]]A-[2'-O-[[2-[2-[[5-[[2-(ACETYLAMINO)-2-DEOXY-BETA-D-GALACTOPYRANOSYL]OXY]-1-OXOPENTYL]AMINO]ETHOXY]ETHOXY]METHYL]]A-GM-GM-CM-UM-GM-CM), COMPLEX WITH RNA ([4'-DE(HYDROXYMETHYL)-4'-[(HYDROXYMETHOXYPHOSPHINYL)METHOXY]]UM-SP-(2'-DEOXY-2'-FLUORO)U-SP-(2'-DEOXY-2'-FLUORO)U-(2'-DEOXY-2'-FLUORO)A-(2'-DEOXY-2'-FLUORO)U-UM-(2'-DEOXY-2'-FLUORO)A-CM-AM-(2'-DEOXY-2'-FLUORO)G-GM-UM-GM-(2'-DEOXY-2'-FLUORO)A-GM-UM-UM-GM-AM-UM-SP-GM-SP-GM)
  • Sponsor:Alexion Pharmaceuticals Inc.

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