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Hemolytic-uremic Syndrome clinical trials

View clinical trials related to Hemolytic-uremic Syndrome.

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NCT ID: NCT05726916 Recruiting - Clinical trials for Hypertensive Emergency-associated Hemolytic Uremic Syndrome

Eculizumab in Hypertensive Emergency-associated Hemolytic Uremic Syndrome

HYPERSHU
Start date: November 9, 2023
Phase: Phase 3
Study type: Interventional

Hemolytic and uremic syndrome (HUS) is a clinic-biological syndrome related to thrombotic microangiopathy affecting predominantly the kidney. Atypical HUS (aHUS) has been historically defined as HUS occurring in the absence of infectious event. The role of complement dysregulation in aHUS pathophysiology has been largely demonstrated, since C genetic rare variants are present in 60-70% aHUS patients. In line with the frequency of C dysregulation in aHUS, Eculizumab, an anti-C5 monoclonal antibody, has dramatically improved aHUS patients prognosis. Numerous conditions have been associated with aHUS, including hypertensive emergency (HE), a syndrome of acute blood pressure flare associated with end-organ damage. In cases of HE-aHUS, whether primary aHUS is complicated by secondary HE, or primary HE leads to secondary aHUS is still debated. The investigators recently demonstrated that C genetic variants frequency was similar in patients with HE-aHUS and patients with aHUS without HE, suggesting a major role for C dysregulation in HE-aHUS. Consequently, the investigators propose to evaluate, in HE-aHUS patients, the benefit of a strategy with early Eculizumab therapy (used within its marketing authorization and its conditions of refunding by the health insurance in usual care), compared to standard of care including tight blood pressure control. The hypothesis suggests that C dysregulation may impact renal prognosis of HE-aHUS patients. The investigator's aim to demonstrate that early Eculizumab therapy improves prognosis of HE-aHUS patients. Method The HYPERSHU study is a randomized, controlled, open-labelled study including HE-aHUS patients with severe AKI and no evidence of other conditions associated with HUS (infections, autoimmunity, drugs, pregnancy). The investigators plan to include 62 patients. Patients will be randomized in 2 arms: - Early Eculizumab therapy (for 3 months) added to standard of care (tight blood pressure control). - Standard of care alone with tight blood pressure control. Renal function after 6 months is the primary evaluation criterium. HE is a frequently associated with aHUS, and strongly impacts patient renal prognosis. Efficient therapeutic strategies are still lacking for this condition. The HYPERSHU study will allow to evaluate the benefit of early Eculizumab therapy in patients with HE-aHUS and severe renal dysfunction.

NCT ID: NCT05684159 Not yet recruiting - Clinical trials for aHUS - Atypical Hemolytic Uremic Syndrome

Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy

Start date: October 2024
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT05569746 Completed - Clinical trials for Pediatric Kidney Disease

A Study to Assess Safety, Efficacy, and Pharmacokinetics of INM004 in Pediatric Patients With STEC-HUS

Start date: October 6, 2022
Phase: Phase 2
Study type: Interventional

Hemolytic Uremic Syndrome (HUS) is a foodborne disease which mainly affects children. It is caused by Escherichia coli bacteria, which release a toxin called Shiga toxin within the body. This infectious form of HUS, defined as STEC-HUS, can cause sporadic cases or outbreaks, as observed in different countries. Argentina has the highest incidence of STEC-HUS worldwide. The disease is endemic, representing approximately 95% of all HUS cases nationwide. STEC-HUS generally begins with diarrhea (with or without blood), and can also cause fever, abdominal pain, and cramps. Then the child may have pallor, altered consciousness, decreased urine output, seizures, and other symptoms. Although death is uncommon (it occurs in 2-4% of cases), it is a very serious disease that mainly affects the kidneys, and also other organs such as the brain. About half of children need to undergo a risky procedure such as dialysis (due to malfunctioning kidneys); and most of them also receive blood transfusions. Around 30% of the patients are left with lifelong consequences that can range from permanent kidney damage to the need for a transplant. So far there is no drug, antibiotic or vaccine to prevent or treat HUS. Current treatment protocols include hospitalization for all patients with HUS, and supportive therapy such as hydration and salt intake. Support therapy is not a specific treatment, but rather helps the body better defend itself against the disease. The purpose of this study is to establish whether it is safe and effective to treat patients who are diagnosed with STEC-HUS, with INM004 (study drug). INM004 is an investigational product "Fraction F(ab')2 of Equine Shiga Antitoxin Immunoglobulin". It is a concentrated and sterile serum obtained from healthy horses immunized against Shiga toxin that contains antibodies capable of neutralizing it. The initial hypothesis is that INM004 would neutralize the entry of Shiga toxin into the body's cells thus preventing the consequent toxic damage. With the proposed treatment, INM004 would eliminate the Shiga toxin, preventing the progression of HUS symptoms and its serious complications (such as the need for and duration of dialysis, duration of hospital stays, as well as neurological, cardiovascular, intestinal complications, among others) which are associated with high morbidity and mortality. This treatment could then have an impact in health costs of STEC-HUS as well as the social costs.

NCT ID: NCT05219110 Recruiting - Clinical trials for Hemolytic-Uremic Syndrome

Hyperhydration in Children With Shiga Toxin-Producing E. Coli Infection

HIKO-STEC
Start date: September 29, 2022
Phase: Phase 3
Study type: Interventional

The objective of this study is to determine if early high volume intravenous fluid administration (hyperhydration) may be effective in mitigating or preventing complications of shiga toxin-producing E. coli (STEC) infection in children and adolescents when compared with traditional approaches (conservative fluid management).

NCT ID: NCT04958265 Recruiting - Clinical trials for Atypical Hemolytic Uremic Syndrome

A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Pediatric Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

COMMUTE-p
Start date: November 17, 2021
Phase: Phase 3
Study type: Interventional

This study aims to evaluate the efficacy and safety of crovalimab in pediatric participants with aHUS.

NCT ID: NCT04889430 Recruiting - Clinical trials for Atypical Hemolytic Uremic Syndrome

Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

APPELHUS
Start date: January 17, 2022
Phase: Phase 3
Study type: Interventional

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.

NCT ID: NCT04861259 Recruiting - Clinical trials for 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)

COMMUTE-a
Start date: October 22, 2021
Phase: Phase 3
Study type: Interventional

This study aims to evaluate the efficacy and safety of crovalimab in adult and adolescent participants with aHUS.

NCT ID: NCT04859608 Active, not recruiting - Clinical trials for Hemolytic-Uremic Syndrome, Atypical

Personalized Spacing of Eculizumab Infusions Based on Therapeutic Pharmacological Monitoring (EspacECU)

EspacECU
Start date: November 1, 2021
Phase: Phase 3
Study type: Interventional

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 high interindividual kinetics variability. A tailored administration of eculizumab based on therapeutic drug monitoring will be compared with real-life administration in adults suffering from an atypical haemolytic uraemic syndrome. The objective is to improve efficiency of eculizumab administration.

NCT ID: NCT04749810 Completed - Clinical trials for Atypical Hemolytic Uremic Syndrome

Observational Study of Elizaria® in aHUS Patients

Start date: December 19, 2019
Phase:
Study type: Observational

It is a multicenter observational non-comparative study of the efficacy and safety of long-term pathogenetic Elizaria® therapy in patients with atypical Hemolytic Uremic Syndrome

NCT ID: NCT04745195 Recruiting - Clinical trials for Thrombotic Microangiopathies

Complement Prospective Evaluation of Thrombotic Microangiopathy on Endothelium

COMPETE
Start date: August 12, 2021
Phase:
Study type: Observational [Patient Registry]

Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions. Dysregulation of the alternative pathway of complement may be the etiological factor and this type of TMA is classified, according to the current nomenclature, as primary atypical hemolytic uremic syndrome (HUS). Half the patients with primary atypical HUS present with rare variants in complement genes, although coexisting conditions are often needed for the TMA to become manifest. In patients with secondary atypical HUS, certain coexisting conditions appear to drive the disease and treatment should target the underlying condition to remit the TMA. Recently, the investigators demonstrated, by using a novel in-house developed functional endothelial cell-based test, that complement dysregulation and overactivation is the dominant cause of disease and its sequelae in a subset of patients with secondary atypical HUS, having impact on treatment and prognosis. The investigators did first prove this concept in patients presenting with TMA and hypertensive emergency. A prospective study is needed to further corroborate these findings along the spectrum of TMA. The investigators hypothesize that their functional endothelial cell-based test, the so-called "HMEC" test, can better categorizes the TMA into different groups with potential therapeutic and prognostic implications. Thus, paving the road to the ultimate goal of precision medicine.