Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)

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What is this study about?

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

Participants in the study will receive Crovalimab and will be monitored over a period of time to see how their condition responds to the treatment. The study will look at various health indicators, such as kidney function and blood parameters, to determine the treatment’s impact. The study will also monitor any side effects or reactions to the treatment, including those at the injection site or related to the immune system.

The study aims to gather information on how well Crovalimab works in managing aHUS and its potential benefits and risks. This information will help in understanding the treatment’s effectiveness and safety for children with this condition. The study is expected to continue until 2029, providing valuable insights into the management of aHUS in pediatric patients.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying vaccination against Neisseria meningitidis within the last three years and ensuring compliance with local guidelines for patients with complement deficiency.

For female patients of childbearing potential, a commitment to abstain from heterosexual intercourse or use contraception is required.

2 treatment initiation

The treatment involves the administration of crovalimab, a solution for injection or infusion. The medication is given through subcutaneous (under the skin) and intravenous (into a vein) routes.

The first dose is administered after confirming the onset of initial TMA (thrombotic microangiopathy) presentation within 28 days prior to the first dose for treatment-naive patients.

3 treatment phase

The treatment phase lasts for 24 weeks. During this period, the effect of crovalimab is monitored through various parameters, including kidney function and blood tests.

Regular assessments are conducted to evaluate the response to treatment, including changes in dialysis requirements, kidney function, and blood parameters.

4 monitoring and evaluation

Throughout the study, the incidence and severity of any adverse events, including injection site reactions and infections, are closely monitored.

The presence of anti-drug antibodies (ADAs) to crovalimab is also assessed to understand the body’s immune response to the medication.

5 completion of treatment

At the end of the 24-week treatment period, a final evaluation is conducted to determine the overall effectiveness of the treatment.

The study aims to measure the proportion of patients achieving a complete TMA response and maintaining TMA control from baseline through Week 25.

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. This is important for patients with a condition called complement deficiency, which affects the immune system.
If the patient is a female who can have children, she must agree to either not have heterosexual intercourse or use birth control during the study.
For patients who have not received treatment before (naive cohort), the first signs of a condition called TMA (a type of blood disorder) must have appeared within 28 days before the first dose of the study medication, crovalimab.
For patients switching from other treatments (switch cohort), there must be clinical evidence showing a response to either eculizumab or ravulizumab. This is shown by a platelet count that is normal or higher, LDH (a type of enzyme) that is normal or lower, and stable or improving kidney function, measured by creatinine levels, in two tests at least 4 weeks apart before starting crovalimab.
For patients in the C5SNP cohort, they must have a known C5 polymorphism (a genetic variation), such as Arg885.
The study is open to both male and female patients.
The study includes patients who are considered a vulnerable population, which means they may need special protection or care.

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 with any other medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who are unable to follow the study procedures or instructions.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within a certain period before this study.
Patients with a history of severe allergic reactions to any of the study medications.
Patients with certain infections that could interfere with the study.
Patients with a history of drug or alcohol abuse that could affect their ability to participate in the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Oncopole Claudius Regaud	Toulouse	France
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Instytut Centrum Zdrowia Matki Polki	Lodz	Poland
Virgen del Rocío University Hospital	Sevilla	Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
CHC MontLegia	Liege	Belgium
Hopital Beaujon	Clichy	France
University Of Szeged	Szeged	Hungary
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Uxkmdbdotpccvz Cmksnqe Krukthzgd	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	30.04.2022
France	Not recruiting	30.04.2022
Hungary	Not recruiting	30.04.2022
Italy	Not recruiting	30.04.2022
Poland	Not recruiting	30.04.2022
Spain	Not recruiting	30.04.2022

Trial locations

Investigated drugs:

Crovalimab

Crovalimab is a medication being studied for its effects on pediatric 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 trial aims to assess how well Crovalimab works, its safety, and how it behaves in the body of patients who have not previously received treatment with complement inhibitors.

Atypical Hemolytic Uremic Syndrome (aHUS) – Atypical Hemolytic Uremic Syndrome is a rare condition characterized by the formation of small blood clots in the blood vessels, which can lead to damage in various organs, particularly the kidneys. It is caused by the abnormal activation of the complement system, a part of the immune system. This disease often results in hemolytic anemia, where red blood cells are destroyed faster than they can be made, leading to fatigue and weakness. Patients may also experience thrombocytopenia, a condition where there are fewer platelets in the blood, increasing the risk of bleeding. Kidney function can be severely affected, potentially leading to acute kidney injury or chronic kidney disease. The progression of aHUS can vary, with some individuals experiencing frequent relapses and others having a more stable course.

Trial ID:

2023-505638-82-00

Protocol code:

BO42354

Trial Phase:

Therapeutic confirmatory (Phase III)

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