Clinical trials: Atypical Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
This study aims to evaluate the efficacy and safety of crovalimab in pediatric participants with aHUS.
- Trial name
- COMMUTE-p (NCT04958265)
- Testing
- Crovalimab
- Where
- 20 sites in United States, Belgium and 8 more
- Volunteers
- 41 people
- Sponsor
- Hoffmann-La Roche
Atypical Hemolytic Uremic Syndrome
Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
This is a multicenter, single arm, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of iptacopan in participants with aHUS.
- Trial name
- NCT05795140
- Testing
- Iptcaopan 200 mg
- Where
- 9 sites in Brazil, China and 4 more
- Volunteers
- 65 people
- Sponsor
- Novartis Pharmaceuticals
Atypical Hemolytic Uremic Syndrome, Membranoproliferative Glomerulonephritis
Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases
This project aims to identify, through RNA-Seq technology, the genetic alterations underlying undiagnosed rare diseases in pediatric and adult patients with early onset and with negative WES.
- Conditions
- Atypical Hemolytic Uremic Syndrome, Membranoproliferative Glomerulonephritis, Autosomal Dominant Polycystic Kidney, Healthy
- Trial name
- ANTHEM (NCT05996731)
- Testing
- Skin biopsy
- Where
- Italy
- Volunteers
- 105 people, healthy volunteers welcome
- Sponsor
- Mario Negri Institute for Pharmacological Research
Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome
Eculizumab to Treat Thrombotic Microangiopathy/Atypical Hemolytic Uremic Syndrome -Associated Multiple Organ Dysfunction Syndrome in Hematopoietic Stem Cell Transplant Recipients
Hematopoietic stem cell transplantation (HCT)-associated thrombotic microangiopathy (TMA) is an understudied complication of HCT that significantly affects transplant related morbidity and mortality.
- Conditions
- Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome, Multiple Organ Dysfunction Syndrome
- Trial name
- NCT03518203
- Testing
- Eculizumab
- Where
- 3 sites in United States
- Volunteers
- 23 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Thrombotic Microangiopathy
OMS721 Compassionate Use in Patients With Thrombotic Microangiopathy
The purpose of this compassionate use study, for two patients with thrombotic microangiopathy, is to provide expanded access to patients who have participated in the clinical trial OMS721-TMA-001 and in whom improvement in their…
- Trial name
- NCT02355782
- Testing
- OMS721
- Sponsor
- Michal Nowicki
Atypical Hemolytic Uremic Syndrome
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Resistant aHUS
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adolescent patients with plasma therapy-resistant Atypical Hemolytic-Uremic Syndrome (aHUS).
- Trial name
- aHUS (NCT00844844)
- Testing
- Eculizumab
- Where
- 17 sites in United States, Austria and 3 more
- Volunteers
- 1 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Atypical Hemolytic Uraemic Syndrome
Complement Activation During Hemodialysis in Atypical Hemolytic Uraemic Syndrome as Underlying Kidney Disease
Atypical hemolytic uraemic syndrome is caused by defects in the regulating factors in the alternative pathway of the complement system.
- Trial name
- NCT00930423
- Where
- Belgium
- Volunteers
- 25 people
- Sponsor
- University Hospital, Ghent
Atypical Hemolytic Uremic Syndrome
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
The purpose of this Phase 3 study is to determine whether iptacopan (LNP023) is efficacious and safe for the treatment of aHUS in adult patients who are treatment naive to complement inhibitor therapy.
- Trial name
- APPELHUS (NCT04889430)
- Testing
- Iptacopan
- Where
- 36 sites in United States, Brazil and 6 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Atypical Hemolytic Uremic Syndrome
The Burden of Atypical Hemolytic Uremic Syndrome and The Clinical Characteristics of Patients in Egyptian Hospitals A Multicenter, Observational, Retrospective Cohort Study in Egypt
Atypical hemolytic uremic syndrome (aHUS) is a rare, progressive, and life-threatening disease that occurs at any age, with incidence rate of 0.75 to 2.0 cases per million population per year.
- Trial name
- REACH (NCT07218536)
- Where
- 6 sites in Egypt
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
Atypical Hemolytic Uremic Syndrome (aHUS)
Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of the study is to assess the safety and efficacy of ravulizumab to control disease activity in adolescent and adult participants with aHUS who had not previously used a complement inhibitor.
- Trial name
- NCT02949128
- Testing
- Ravulizumab
- Where
- 37 sites in United States, Australia and 12 more
- Volunteers
- 58 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic Syndrome
Complement Inhibition in aHUS Dialysis Patients
This study evaluates the effect of CCX168, a C5aR Antagonist, Oral Administration on Ex Vivo Thrombus Formation and Disease Activity in ten patients with diagnosis of Atypical Hemolytic Uremic Syndrome with or without genetic…
- Trial name
- ACCESS (NCT02464891)
- Testing
- CCX168
- Where
- Italy
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Mario Negri Institute for Pharmacological Research
Atypical Hemolytic Uremic Syndrome
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
- Trial name
- NCT03303313
- Testing
- Cemdisiran
- Where
- 12 sites in Bosnia and Herzegovina, Canada and 8 more
- Sponsor
- Alnylam Pharmaceuticals
Atypical Hemolytic-Uremic Syndrome
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.
- Trial name
- NCT01522183
- Where
- 141 sites in United States, Australia and 15 more
- Volunteers
- 3,000 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome
TTP and aHUS in Complicated Pregnancies
A single site observational study aiming to: (i) Identify cases of previously undiagnosed thrombotic thrombocytopenic purpura (TTP) and atypical haemolytic syndrome (aHUS) in a cohort of women with complicated pregnancies (ii)…
- Conditions
- Thrombotic Thrombocytopenic Purpura, Atypical Hemolytic Uremic Syndrome, Pre-Eclampsia, HELLP Syndrome
- Trial name
- NCT03605511
- Testing
- ADAMTS 13, complement and angiogenic biomarkers
- Where
- United Kingdom
- Volunteers
- 150 people
- Sponsor
- University College, London
Atypical Hemolytic-Uremic Syndrome
An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
The primary purpose is to assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
- Trial name
- aHUS (NCT01193348)
- Testing
- Eculizumab
- Where
- 17 sites in United States, Australia and 7 more
- Volunteers
- 22 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Atypical Hemolytic Uremic Syndrome
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
This study aims to evaluate the efficacy and safety of crovalimab in adult and adolescent participants with aHUS.
- Trial name
- COMMUTE-a (NCT04861259)
- Testing
- Crovalimab
- Where
- 41 sites in United States, Belgium and 14 more
- Volunteers
- 83 people
- Sponsor
- Hoffmann-La Roche
Atypical Hemolytic Uremic Syndrome
Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS
Atypical hemolytic syndrome (aHUS) is a severe renal disease affecting children and adults. It is characterized by the occlusion of intrarenal vessels due to the presence of platelet/fibrin thrombi, and leads to end-stage renal…
- Trial name
- STOPECU (NCT02574403)
- Testing
- eculizumab
- Where
- 22 sites in France
- Volunteers
- 58 people
- Sponsor
- Nantes University Hospital
Atypical Hemolytic Uremic Syndrome
Ravulizumab Outcomes in Polish Patients With aHUS
This multicenter, observational cohort study uses retrospective collection of past medical history and prospective follow-up to capture longitudinal data on the management and clinical outcomes of patients with atypical hemolytic…
- Trial name
- aHUS-OPTIMUM (NCT07399730)
- Testing
- Ravulizumab
- Where
- 10 sites in Poland
- Volunteers
- 80 people
- Sponsor
- AstraZeneca