Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)

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What is this study about?

This clinical trial is focused on studying atypical Hemolytic Uremic Syndrome (aHUS), a rare disease that affects the blood and kidneys. The study will evaluate a treatment called Crovalimab, which is administered as a solution for injection or infusion. The purpose of the study is to assess how effective and safe Crovalimab is for patients who have not previously received treatment that inhibits a part of the immune system known as the complement system.

Participants in the study will receive Crovalimab through injections under the skin or infusions into a vein. The study will monitor various health indicators, such as kidney function and blood health, to determine the treatment’s impact. The study will also track any side effects or reactions to the treatment, including changes in fatigue levels and any adverse events that may occur.

The trial aims to understand how well Crovalimab works in controlling the symptoms of aHUS and improving patients’ overall health. The study will gather information over a period of time to see how patients respond to the treatment and whether it helps maintain control over the disease. This research is important for developing new treatment options for people living with aHUS.

1 initial assessment

The study begins with an initial assessment to confirm eligibility. This includes ensuring vaccination against Neisseria meningitidis within the last three years, adequate liver function, and for women of childbearing potential, a commitment to abstinence or contraception.

For patients new to treatment, the onset of initial symptoms must have occurred within 28 days before the first dose of crovalimab.

2 treatment initiation

The treatment involves the administration of crovalimab, which is a solution for injection or infusion. It is administered either subcutaneously or intravenously, depending on the specific requirements of the study.

The objective is to evaluate the effect of crovalimab in patients who have not previously received complement inhibitor treatment.

3 monitoring and evaluation

Throughout the study, various health parameters are monitored. This includes the requirement for dialysis, changes in kidney function, blood parameters, and fatigue levels in adults.

The study also tracks the incidence and severity of any adverse events, including reactions at the injection site, infusion-related reactions, and infections.

4 response assessment

The primary goal is to determine the proportion of patients achieving a complete response to treatment. This involves monitoring platelet counts, lactate dehydrogenase (LDH) levels, and serum creatinine levels.

Secondary assessments include changes in chronic kidney disease stage, vital signs, and the presence of anti-drug antibodies.

5 study completion

The study is estimated to conclude by December 7, 2029. During this period, the effectiveness and safety of crovalimab will be thoroughly evaluated.

The study aims to provide comprehensive data on the long-term management of atypical hemolytic uremic syndrome (aHUS) with crovalimab.

Who Can Join the Study?

The patient must have been vaccinated against Neisseria meningitidis (a type of bacteria) within the last 3 years before starting the study treatment, following local guidelines or standard care for patients with complement deficiency (a condition where part of the immune system is not working properly).
The patient must have adequate liver function, with AST and ALT (liver enzymes) levels no more than 3 times the upper limit of normal at the time of screening. There should be no clinical signs or known evidence of cirrhosis (severe liver damage).
Female patients who can have children must agree to either not have heterosexual intercourse or use contraception.
For patients who have not received complement inhibitor treatment before, the first signs of TMA (a condition affecting blood and kidneys) must have appeared within 28 days before the first dose of the study drug, crovalimab.
For patients switching from other treatments, there must be clinical evidence of response to either eculizumab or ravulizumab, shown by a platelet count at or above the lower limit of normal, LDH (an enzyme) at or below the upper limit of normal, and stable or improving kidney function, measured at two separate times at least 4 weeks apart before starting crovalimab treatment.
For patients with a known C5 polymorphism (a genetic variation), such as Arg885, they may be eligible for a specific group in the study.
The study is open to both male and female patients.
The study includes patients from vulnerable populations.

Who Cannot Join the Study?

Patients who have previously received treatment with a complement inhibitor. A complement inhibitor is a type of medication that affects a part of the immune system called the complement system.
Patients who are not within the specified age range for the study. The study includes specific age groups, so patients must fall within these age categories to participate.
Patients who have any medical condition that the study doctors believe would make it unsafe for them to participate. This could include other serious health problems.
Patients who are unable to follow the study procedures or attend the required study visits. This means they must be able to understand and agree to the study requirements.
Patients who are pregnant or breastfeeding. This is to ensure the safety of both the mother and the baby.
Patients who are participating in another clinical trial at the same time. This is to avoid any interference with the study results.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Medizinische Hochschule Hannover	Hanover	Germany
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Hospital Universitario 12 De Octubre	Madrid	Spain
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego	Warsaw	Poland
Virgen del Rocío University Hospital	Sevilla	Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Hospital Clinic De Barcelona	Barcelona	Spain
IRCCS Ospedale Policlinico San Martino	Genoa	Italy
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet	Budapest	Hungary
Hopital Beaujon	Clichy	France
Uujpblcquzerbkzjhbomr Elwuc Azb	Essen	Germany
Spquctmtnnw Pksjclxug Ssleywk Kdjgnbvai Nm 1 Iufleaebqrixabzate Shlgjwc Sccqoaipq Uqrggcqtsghp Mpxkteifhw W Karomlioim	Zabrze	Poland
Ukexdlqurq Hqvqjdpg Cxptsqc	Cologne	Germany
Unwinuykpadymd Ckhinuq Ksxnjqhmv	Gdansk	Poland
Hmvghxcj Uzrwrzmzaorct do A Cbigsh	A Coruna Galicia	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	19.11.2021
France	Not recruiting	19.11.2021
Germany	Not recruiting	19.11.2021
Hungary	Not recruiting	19.11.2021
Italy	Not recruiting	19.11.2021
Poland	Not recruiting	19.11.2021
Spain	Not recruiting	19.11.2021

Trial locations

Investigated drugs:

Crovalimab

Crovalimab is a medication being studied for its effects on patients with atypical Hemolytic Uremic Syndrome (aHUS). This condition is a rare disease that affects the blood and kidneys. Crovalimab works by inhibiting a part of the immune system called the complement system, which is thought to play a role in the development of aHUS. The study aims to evaluate how effective and safe Crovalimab is for patients who have not previously received treatment with complement inhibitors.

Atypical Hemolytic Uremic Syndrome (aHUS) – Atypical Hemolytic Uremic Syndrome is a rare disease that affects the blood and blood vessels. It is characterized by the destruction of red blood cells, low platelet count, and kidney failure due to damage to the small blood vessels in the kidneys. The disease can lead to the formation of blood clots in small blood vessels, which can cause damage to various organs. aHUS is often triggered by an abnormal activation of the immune system, specifically the complement system, which is part of the body’s defense against infections. The progression of the disease can vary, with some individuals experiencing sudden and severe symptoms, while others may have a more gradual onset. It is important to monitor kidney function and blood parameters in individuals with aHUS to manage the disease effectively.

Trial ID:

2023-505089-27-00

Protocol code:

BO42353

Trial Phase:

Therapeutic confirmatory (Phase III)

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