Clinical trials: sponsored by Alexion Pharmaceuticals, Inc.
Healthy Adult Participants
Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of ALXN2230 in Healthy Participants
The primary objective of this study is to evaluate the safety and tolerability of single subcutaneous (SC) doses of ALXN2230 in healthy participants.
- Trial name
- NCT07352423
- Testing
- ALXN2230
- Where
- United Kingdom
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Registry of Patients With Hypophosphatasia
In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.
- Trial name
- NCT02306720
- Where
- 67 sites in United States, Australia and 11 more
- Volunteers
- 1,571 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.
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis
The primary objective of this study is to evaluate the safety and tolerability of tarperprumig in participants with newly diagnosed or relapsing anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
- Trial name
- NCT07160608
- Testing
- Tarperprumig
- Where
- 57 sites in Argentina, Australia and 12 more
- Volunteers
- 78 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Chronic Kidney Disease, CKD
ARTEMIS: Ravulizumab to Protect Patients With CKD From CSA-AKI and MAKE
The primary objective of this study is to assess the efficacy of a single dose of ravulizumab IV compared with placebo in reducing the risk of the clinical consequences of AKI (MAKE) at 90 days in adult participants with CKD who…
- Conditions
- Chronic Kidney Disease, CKD, Cardiac Disease, Cardiopulmonary Bypass
- Trial name
- ARTEMIS (NCT05746559)
- Testing
- Ravulizumab
- Where
- 130 sites in United States, Argentina and 18 more
- Volunteers
- 555 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Transthyretin Amyloid Cardiomyopathy
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total…
- Trial name
- DepleTTR-CM (NCT06183931)
- Testing
- ALXN2220
- Where
- 213 sites in United States, Argentina and 25 more
- Volunteers
- 1,182 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis, gMG
Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)
The primary purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese adult participants with Acetylcholine receptor (AChR) + Generalized…
- Trial name
- NCT07596784
- Testing
- Ravulizumab
- Where
- 10 sites in China
- Volunteers
- 20 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Neuromyelitis Optica Spectrum Disorder, NMOSD
Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
Long-term, multicenter, multinational, observational, registry of patients with AQP4+ NMOSD that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT).
- Conditions
- Neuromyelitis Optica Spectrum Disorder, NMOSD, AQP4+ NMOSD
- Trial name
- NCT05966467
- Where
- 33 sites in United States, Argentina and 6 more
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Neuromyelitis Optica
Study of the Clinical and Radiological Impact of Ravulizumab in People With Neuromyelitis Optica Spectrum Disorder
This is an observational study to: evaluate the on-treatment clinical performance of ravulizumab in relation to the pre-treatment time period (time period prior to exposure), enhance knowledge regarding conventional MRI outcomes…
- Trial name
- AMAZE (NCT06398158)
- Testing
- Ravulizumab
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- University of Texas Southwestern Medical Center
Amyloid Transthyretin Cardiomyopathy
Study of Re-Treatment With ALXN2220 in Patients With ATTR-CM
This is a Phase 2, single-arm, multicenter study to evaluate the pharmacodynamics and safety of re-treatment with ALXN2220 in previous participants of Study NI006-101.
- Trial name
- NCT07213583
- Testing
- ALXN2220
- Where
- 6 sites in France, Germany and 2 more
- Volunteers
- 35 people
- Sponsor
- Neurimmune AG
Immunoglobulin A Nephropathy, IgAN
Study of Ravulizumab in Immunoglobulin A Nephropathy (IgAN)
The primary objective of this study to evaluate efficacy of ravulizumab compared with placebo on proteinuria reduction and change in eGFR in adult participants with IgAN who are at risk of disease progression.
- Trial name
- I CAN (NCT06291376)
- Testing
- Ravulizumab
- Where
- 246 sites in United States, Argentina and 26 more
- Volunteers
- 584 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, Embolic Stroke of Undetermined Source
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal hematological disorder, which can cause arterial or venous thrombosis.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Cerebral Vein Thrombosis
- Trial name
- NCT03329365
- Where
- Canada
- Volunteers
- 200 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
NMOSD, Neuromyelitis Optica Spectrum Disorder
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity Study of Ravulizumab in Chinese Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)
The primary objective of this study is to confirm the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of ravulizumab in the treatment of Chinese adults with anti-aquaporin-4 (AQP4) antibody (Ab)…
- Trial name
- NCT07557420
- Testing
- Ravulizumab
- Where
- 10 sites in China
- Volunteers
- 19 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
BAG3 Mutation Associated Dilated Cardiomyopathy
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
- Trial name
- DCMRestore (NCT07218887)
- Testing
- ALXN2350
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
The purpose of Study ALXN2220-ATTRCM-302 is to evaluate the long-term safety, tolerability and efficacy of ALXN2220 in adult participants with ATTR-CM treated with ALXN2220 in addition to SoC who have completed Study…
- Trial name
- LTSAFE-ALXN (NCT07789483)
- Testing
- ALXN2220
- Where
- 195 sites in United States, Argentina and 25 more
- Volunteers
- 1,180 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Antibody-Mediated Rejection, Kidney Transplantation
Phase 2 Study of ALXN2030 in Patients With Antibody-Mediated Rejection After Kidney Transplantation
The primary objective of this study is to evaluate the efficacy of ALXN2030 compared with placebo on biopsy proven histologic resolution in participants with active or chronic active antibody-mediated rejection (AMR) at Week 52.
- Conditions
- Antibody-Mediated Rejection, Kidney Transplantation, Biopsy-proven Histologic Scores, AMR
- Trial name
- CONCORD (NCT06744647)
- Testing
- ALXN2030
- Where
- 51 sites in United States, Brazil and 6 more
- Volunteers
- 47 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acromegaly
ALXN2420 Versus Placebo in Combination With Somatostatin Analogs in Participants With Acromegaly
The primary objective of this study is to evaluate the efficacy of 15-week treatment with ALXN2420 versus placebo for decreasing insulin-like growth factor IGF-1 levels, when administered in combination with somatostatin analog…
- Trial name
- ASTERIA (NCT07037420)
- Testing
- ALXN2420
- Where
- 6 sites in United States, Italy and 2 more
- Volunteers
- 8 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
IgAN, IgAVN
Study of Ravulizumab in Pediatric Participants With Primary IgAN
The primary objectives of this study are to characterize ravulizumab pharmacokinetics (PK) and pharmacodynamics (PD), and to evaluate safety and efficacy following ravulizumab IV dosing in pediatric participants with IgAN or…
- Conditions
- IgAN, IgAVN, Immunoglobulin A Nephropathy, Immunoglobulin A Vasculitis Associated Nephritis
- Trial name
- NCT07024563
- Testing
- Ravulizumab
- Where
- 15 sites in United States, China and 5 more
- Volunteers
- 36 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant
A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and…
- Trial name
- ALX-TMA-502 (NCT07732361)
- Where
- 43 sites in United States, Belgium and 8 more
- Volunteers
- 307 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine