Clinical trials: Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy
Hemolytic Uremic Syndrome (HUS) is a rare disease characterized by rupture of red blood cells (hemolytic anemia), low platelet count (thrombocytopenia), and thrombotic occlusion of small vessels (thrombotic microangiopathy), with…
- Trial name
- aHUS-iPSC-EC (NCT05805202)
- Testing
- Blood sampling and urine analysis
- Where
- Italy
- Volunteers
- 112 people, healthy volunteers welcome
- Sponsor
- Mario Negri Institute for Pharmacological Research
Hemolytic-Uremic Syndrome, Membranoproliferative Glomerulonephritis
New Analytic Tools for aHUS and C3G Diagnosis
This protocol is part of a larger project, COMPRare (COMPlement-mediated Rare kidney diseases), which has been financed on behalf of the EJP RD (European Joint Programme on Rare Diseases) program of EU and is leaded by a…
- Conditions
- Hemolytic-Uremic Syndrome, Membranoproliferative Glomerulonephritis, Healthy
- Trial name
- COMPRare (NCT05985122)
- Testing
- C3NEF assay
- Where
- Italy
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Mario Negri Institute for Pharmacological Research
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
Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS
The purpose of this study is to determine whether eculizumab is safe and effective in the treatment of adolescent patients with plasma therapy-sensitive Atypical Hemolytic-Uremic Syndrome (aHUS).
- Trial name
- aHUS (NCT00844428)
- Testing
- eculizumab
- Where
- 13 sites in Canada, France and 5 more
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Bloody Diarrhea, STEC, Unspecified
Phase 2/3 Study to Evaluate PK, Safety & Efficacy of INM004 in STEC Positive Pediatric Patients for Prevention of HUS
The investigational medicinal product (IMP), INM004, proposes to neutralize the toxin in the bloodstream to prevent the interaction of the Stx with the specific receptor, by means of a polyclonal antibody to be administered upon…
- Conditions
- Bloody Diarrhea, STEC, Unspecified, Hemolytic-Uremic Syndrome, Pediatric Kidney Disease
- Trial name
- NCT04132375
- Testing
- INM004
- Where
- 7 sites in Argentina
- Volunteers
- 11 people
- Sponsor
- Inmunova S.A.
Hemolytic-Uremic Syndrome, Atypical
Personalized Spacing of Eculizumab Infusions Based on Therapeutic Pharmacological Monitoring (EspacECU)
Eculizumab is an anti-C5 monoclonal antibody approved for rare diseases including atypical haemolytic-uraemic syndrome. The maintenance phase dosing regimen is identical for all adult patients but several studies have shown a…
- Trial name
- EspacECU (NCT04859608)
- Testing
- Spacing of Eculizumab infusions
- Where
- 18 sites in France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Tours
aHUS - Atypical Hemolytic Uremic Syndrome
Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy
This is a Phase II, open-label study designed to determine if intravenously administered NM8074 results in remission from TMA in treatment-naïve aHUS patients.
- Trial name
- NCT05684159
- Testing
- NM8074
- Volunteers
- 12 people
- Sponsor
- NovelMed Therapeutics
Hemolytic Uremic Syndrome
Study of 'Vascular Competence' Profile and Endothelial Activation in the Hemolytic Uremic Syndrome in Children and Adults
The Hemolytic Uremic Syndrome (HUS) is a rare thrombotic microangiopathy (TMA), affecting both children and adults. HUS is characterized by the abnormal occurrence of diffuse thrombosis in the microcirculation resulting in the…
- Trial name
- SHU (NCT02904863)
- Testing
- Extra blood draw samples
- Where
- France
- Volunteers
- 19 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
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 (AHUS)
To Characterize the Safety and Tolerability of Eculizumab in Two Japanese aHUS Patients
This protocol is intended to formally collect data on the treatment of aHUS with eculizumab in Japanese patients.
- Trial name
- NCT01755429
- Where
- 2 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Mucositis, Graft-versus-host Disease
Tacrolimus and Mycophenolate Mofetil (MMF) in GVHD Prophylactic Regimen Compared to Tacrolimus and Methotrexate (MTX
A comparative trial where all patients will receive daily doses of tacrolimus (TAC) until day +60 when tapering will begin, in the absence of graft-versus-host disease (GVHD), and discontinued by day +180.
- Trial name
- NCT00360685
- Testing
- Tac+MTX, TAC + MMF
- Where
- United States
- Volunteers
- 89 people
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Hemolytic-Uremic Syndrome
Efficacy of INM004 in Children With STEC-HUS
The objectives of this study are to evaluate the efficacy, safety, and pharmacokinetics of INM004 in pediatric patients with Hemolytic Uremic Syndrome associated to infection by Shiga toxin-producing Escherichia coli (STEC-HUS).
- Trial name
- NCT06389474
- Testing
- INM004
- Where
- 52 sites in Argentina, Belgium and 7 more
- Volunteers
- 220 people
- Sponsor
- Inmunova S.A.
Atypical Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura
The Role of Microparticles as a Biomarker
The investigators propose to characterize MPs in aHUS and TTP both at the onset and throughout treatment. The investigators believe that the number, size, and cell origin of MPs will differ between these two diseases.
- Conditions
- Atypical Hemolytic Uremic Syndrome, Thrombotic Thrombocytopenic Purpura, Microparticles, Microangiopathic Hemolytic Anemia
- Trial name
- NCT02626663
- Where
- United States
- Sponsor
- University of Rochester
Atypical Hemolytic Uremic Syndrome (aHUS)
Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)
The purpose of the study is to assess the efficacy of ravulizumab to control disease activity in children and adolescents with aHUS who have not previously used a complement inhibitor (complement inhibitor treatment-naïve), as…
- Trial name
- NCT03131219
- Testing
- Ravulizumab
- Where
- 18 sites in United States, Belgium and 5 more
- Volunteers
- 34 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
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.
Atypical Hemolytic Uremic Syndrome(aHUS)
A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients
This is a China, non-interventional, observational study and will follow the Good Phar-macoepidemiology Practices guidelines. This study will enrol paediatric and adult patients diagnosed with aHUS who will be treated according…
- Trial name
- NCT06099236
- Where
- 26 sites in China
- Volunteers
- 367 people
- Sponsor
- AstraZeneca