Clinical trials: sponsored by Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.
- Trial name
- HICKORY (NCT06079281)
- Testing
- ALXN1850
- Where
- 65 sites in United States, Argentina and 15 more
- Volunteers
- 124 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Primary Membranous Nephropathy
Study of ALXN1920 in Adult Participants With Primary Membranous Nephropathy (PMN)
The primary objective of this study is to evaluate the efficacy of ALXN1920 compared with placebo in participants with PMN who are at a high risk for disease progression using 24-hour urine protein creatinine ratio (UPCR).
- Trial name
- AUTUMN (NCT07157787)
- Testing
- ALXN1920
- Where
- 40 sites in United States, Argentina and 8 more
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Delayed Graft Function, DGF
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
The primary objective of this study is to demonstrate the efficacy of ravulizumab vs placebo in reducing the severity of DGF as measured by time to freedom from dialysis in adult participants who are at high risk of DGF after…
- Conditions
- Delayed Graft Function, DGF, Kidney Transplant
- Trial name
- AWAKE (NCT06830798)
- Testing
- Ravulizumab
- Where
- 130 sites in United States, Argentina and 16 more
- Volunteers
- 450 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Microangiopathy
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
- Trial name
- NCT04543591
- Testing
- Ravulizumab, Best supportive care
- Where
- 66 sites in United States, Australia and 15 more
- Volunteers
- 148 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Trial name
- NCT05389449
- Testing
- Danicopan
- Where
- 45 sites in United States, Brazil and 13 more
- Volunteers
- 80 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
AL Amyloidosis
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down.
- Trial name
- NCT04512235
- Testing
- CAEL-101, cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
- Where
- 110 sites in United States, Australia and 17 more
- Volunteers
- 281 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis
Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis
The purpose of this study is to evaluate the safety and efficacy of ALXN1720 for the treatment of generalized MG (gMG) in adults with autoantibodies against acetylcholine receptor (AChR).
- Trial name
- NCT05556096
- Testing
- ALXN1720
- Where
- 132 sites in United States, Argentina and 20 more
- Volunteers
- 260 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged.
- Trial name
- NCT05981092
- Where
- 15 sites in United States, Netherlands and 3 more
- Volunteers
- 35 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Symptomatic Transthyretin Amyloid Cardiomyopathy
A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
This prospective study is designed to evaluate the efficacy, safety, and tolerability of ALXN2060 (also known as AG10), as well as to establish its pharmacokinetic and pharmacodynamic profile in Japanese participants with…
- Trial name
- NCT04622046
- Testing
- ALXN2060
- Where
- 11 sites in Japan
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
- Trial name
- NCT06578949
- Testing
- Ravulizumab
- Where
- 8 sites in China
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Danicopan Early Access Program
This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT05982938
- Testing
- Danicopan
- Where
- 15 sites in Italy
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal…
- Trial name
- Soliris (NCT05886244)
- Testing
- Eculizumab
- Where
- 7 sites in China
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.
- Trial name
- MULBERRY (NCT06079359)
- Testing
- ALXN1850
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical…
- Trial name
- NCT05274633
- Testing
- Ravulizumab
- Where
- 6 sites in Italy
- Volunteers
- 120 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.
- Trial name
- CHESTNUT (NCT06079372)
- Testing
- ALXN1850, asfotase alfa
- Where
- 21 sites in United States, Argentina and 5 more
- Volunteers
- 43 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
aHUS, Atypical Hemolytic Uremic Syndrome
Post-Marketing Clinical Study of Ravulizumab in Participants With Clinical aHUS
The primary objective of this study is to assess the platelet count response to ravulizumab in participants clinically diagnosed as atypical hemolytic uremic syndrome (aHUS).
- Trial name
- NCT07308574
- Testing
- Ravulizumab
- Where
- 12 sites in Japan
- Volunteers
- 20 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis, gMG
Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
- Trial name
- NCT05644561
- Testing
- Ravulizumab
- Where
- 11 sites in United States, Italy and 3 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis, gMG
A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab
This is a prospective, multicenter single arm study designed to evaluate the effectiveness and safety of a predefined oral corticosteroids (OCS) tapering schedule to reduce OCS use in adult participants with acetylcholine…
- Trial name
- OCTAGON (NCT07221838)
- Testing
- Oral Corticosteroid Tapering Schedule, Ravulizumab, Prednisone/Prednisolone
- Where
- 12 sites in United States, Germany and 2 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
Safety, PK/PD, and Immunogenicity Study of SC ALXN2030 in Healthy Participants
The primary purpose of this study is to assess the safety and tolerability of single ascending doses of ALXN2030 in healthy participants.
- Trial name
- NCT05501717
- Testing
- ALXN2030
- Where
- United Kingdom
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.