Clinical trials: sponsored by 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.
Generalized Myasthenia Gravis, gMG
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
- Trial name
- NCT06607627
- Testing
- Gefurulimab
- Where
- 13 sites in United States, Brazil and 3 more
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy
Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants
The purpose of this study is to determine the effect of a high-fat, high-caloric meal on the pharmacokinetics (PK) of acoramidis in healthy adult participants following an oral single dose administration of acoramidis.
- Trial name
- NCT04958135
- Testing
- Acoramidis
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
Lysosomal Acid Lipase (LAL) Deficiency Registry
This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency.
- Conditions
- Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease, Wolman Disease, Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
- Trial name
- ALX-LALD-501 (NCT01633489)
- Where
- 104 sites in United States, Australia and 20 more
- Volunteers
- 300 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis
Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)
Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab…
- Trial name
- NCT04202341
- Where
- 51 sites in United States, Austria and 5 more
- Volunteers
- 500 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data
This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry.
- Trial name
- NCT07413250
- Testing
- Danicopan, Soliris/Ultomiris
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Long-term Safety of Danicopan: IPIG Registry-based Cohort Study
This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry.
- Trial name
- NCT07413679
- Testing
- Danicopan
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
A Prospective Sub-Study of the Global Hypophosphatasia Registry
In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and…
- Trial name
- NCT05234567
- Testing
- Asfotase Alfa
- Where
- 12 sites in United States
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis, Anti-AChR Antibody Positive
Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis
The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.
- Trial name
- NCT06967480
- Testing
- Ravulizumab
- Where
- 23 sites in Italy
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Methylmalonic Acidemia
Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
This is a non-interventional long-term follow-up study of patients who have participated in LogicBio studies for the treatment of MMA and received hLB-001
- Trial name
- NCT05506254
- Testing
- hLB-001
- Where
- 4 sites in United States
- Volunteers
- 4 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Lupus Nephritis, Immunoglobulin A Nephropathy
Study of Ravulizumab in Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
The objectives of this study are to evaluate the safety and efficacy of ravulizumab administered by intravenous (IV) infusion compared to placebo and demonstrate proof-of-concept of the efficacy of terminal complement inhibition…
- Trial name
- SANCTUARY (NCT04564339)
- Testing
- Ravulizumab, Background Therapy
- Where
- 64 sites in United States, Australia and 10 more
- Volunteers
- 123 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT01374360
- Where
- United States
- Volunteers
- 6,061 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Generalized Myasthenia Gravis (gMG), Refractory gMG
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With gMG
This is an open-label, single-arm, multi-center study to evaluate the efficacy, safety, PK, PD, and immunogenicity of eculizumab in Chinese participants with refractory gMG.
- Trial name
- ECU-MG-304 (NCT06764160)
- Testing
- Eculizumab
- Where
- 6 sites in China
- Volunteers
- 15 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Microangiopathy
Study of Ravulizumab in Pediatric Participants With HSCT-TMA
This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA.
- Trial name
- NCT04557735
- Testing
- Ravulizumab, Best Supportive Care
- Where
- 26 sites in United States, Israel and 5 more
- Volunteers
- 41 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Transthyretin-type Cardiac Amyloidosis
Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
- Trial name
- ATTR-CM (NCT07306949)
- Testing
- Acoramidis
- Where
- 16 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT06449001
- Testing
- Danicopan
- Where
- 4 sites in Canada, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
NMOSD, Neuromyelitis Optica Spectrum Disorders
Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD
The primary objective of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of Eculizumab in Chinese Adults with Neuromyelitis Optica Spectrum Disorders (NMOSD).
- Trial name
- ECU-NMO-304 (NCT06724809)
- Testing
- eculizumab
- Where
- 7 sites in China
- Volunteers
- 21 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for Hypophosphatasia
The primary objective of this study is to evaluate the effect of immunosuppressive therapy (IST) in participants treated with asfotase alfa who demonstrate immune-mediated loss of effectiveness (LoE).
- Trial name
- RESTORE (NCT06015750)
- Testing
- methotrexate, rituximab, bortezomib
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic
Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China
This is a Phase 3b, open-label, single-arm, multicenter study to evaluate the efficacy and safety of eculizumab in participants with atypical hemolytic uremic syndrome (aHUS) in China
- Trial name
- Soliris (NCT05876351)
- Testing
- Eculizumab
- Where
- 6 sites in China
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pemphigoid, Bullous
Evaluation of Safety, Efficacy and Pharmacodynamic Effect of Bertilimumab in Patients With Bullous Pemphigoid
This is an open-label, proof-of-concept, single group study in adult participants with newly diagnosed, moderate to extensive BP.
- Trial name
- NCT02226146
- Testing
- Bertilimumab
- Where
- 8 sites in United States, Israel
- Volunteers
- 11 people
- Sponsor
- Alexion Pharmaceuticals, Inc.