RecruitingRare disease

Study on Ravulizumab for Children with IgA Nephropathy (Ages 2-17)

Verified siteRegistered drugNo placebo

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a kidney disease called Primary Immunoglobulin A Nephropathy (IgAN) in children aged 2 to under 18 years. The treatment being tested is a medication called Ravulizumab, which is given through an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein. Ravulizumab is also known by its code name, ALXN1210.

The purpose of the study is to understand how Ravulizumab works in the body and how safe and effective it is for treating IgAN in children. The study will involve giving the medication to participants and monitoring them over a period of time to see how their bodies respond to the treatment. This will help researchers learn more about the medication's effects and how it can be used to treat this kidney disease in children.

Participants in the study will receive the medication and be observed by healthcare professionals to ensure their safety and to gather information about the treatment's impact. The study aims to provide valuable insights that could help improve treatment options for children with IgAN in the future.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit
Upon joining the study, the participant will attend an initial visit. During this visit, the participant's medical history will be reviewed, and a physical examination will be conducted to ensure eligibility for the trial.
The participant will receive information about the study, including the purpose, procedures, and potential risks and benefits. Informed consent will be obtained from the participant's legal guardian, and assent will be obtained from the participant if applicable.
Step 2
Screening period
During the screening period, the participant will undergo various tests to confirm eligibility. These tests may include blood and urine tests, as well as a review of current medications.
The participant must have a confirmed diagnosis of primary immunoglobulin A nephropathy (IgAN) based on a kidney biopsy obtained prior to the first day of the study.
Step 3
Vaccination
To reduce the risk of infection, the participant must be vaccinated against meningococcal infection from serogroups A, C, W135, and Y, and serogroup B if available, at least two weeks before the first dose of the study medication.
Vaccination against Haemophilus influenzae type b and Streptococcus pneumoniae is also required unless previously completed according to national and local guidelines.
Step 4
Treatment phase
The participant will receive the study medication, ravulizumab, administered as an intravenous infusion. The dosage and frequency will be determined by the study protocol and the participant's weight.
The treatment phase will continue for a specified duration, with regular visits to monitor the participant's response to the medication and any side effects.
Step 5
Follow-up visits
Throughout the study, the participant will attend follow-up visits to assess the effectiveness and safety of the treatment. These visits will include physical examinations, blood tests, and other assessments as required by the study protocol.
The participant's legal guardian will be responsible for maintaining a take-home record of the participant's general health and any changes observed during the study.
Step 6
End of study
At the end of the study, a final assessment will be conducted to evaluate the participant's overall health and the outcomes of the treatment.
The participant and their legal guardian will receive information about the study's findings and any further steps that may be necessary for the participant's health care.

Who can join the trial?

13 criteria

Participant must be between the ages of 2 and 17 years old at the time of signing the consent form.
The legal guardian must provide written consent, and the child must agree to participate if they are able to understand.
The legal guardian or primary caregiver must be able to keep an accurate record of the child's health at home.
The child must have a confirmed diagnosis of Immunoglobulin A Nephropathy (IgAN) or IgA Vasculitis Nephritis (IgAVN) based on a kidney biopsy done before the study starts.
If the child is taking medications to suppress the immune system (like corticosteroids or other specific drugs) for kidney issues related to IgAVN, the medication must be stable for at least 1 month before the study begins.
The child must have a urine protein to creatinine ratio (UPCR) of at least 0.5 g/g from samples collected at home within 1 week during the screening period.
The child must have an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m² during screening, which is a measure of kidney function.
The child must be on a stable dose of Renin-Angiotensin System Inhibitors (RASI) like ACE inhibitors or ARBs for at least 3 months before screening, with no planned changes in dose during the study. Children who cannot tolerate these medications may still participate.
The child must weigh at least 10 kg (about 22 pounds) at the time of screening.
Both boys and girls can participate, regardless of their gender identity.
Appropriate contraceptive measures and pregnancy testing must be followed based on the child's age and local regulations.
To reduce the risk of meningococcal infection, the child must be vaccinated against meningococcal bacteria (types A, C, W135, Y, and B if available) within the last 3 years and at least 2 weeks before the first dose of the study drug. If vaccinated less than 2 weeks before the study drug, the child will need antibiotics for at least 2 weeks after vaccination.
The child must have received vaccinations against Haemophilus influenzae type b and Streptococcus pneumoniae unless they have already been vaccinated according to current guidelines.

Who cannot join the trial?

10 criteria

Patients with a history of severe allergic reactions to the study medication cannot participate.
Patients who are currently participating in another clinical trial are not eligible.
Patients with uncontrolled high blood pressure are excluded.
Patients with active infections that require treatment with antibiotics are not allowed to join.
Patients with a history of certain types of cancer may not participate.
Patients who have received an organ transplant are excluded.
Patients with severe liver disease cannot take part in the study.
Patients who are pregnant or breastfeeding are not eligible.
Patients with a history of drug or alcohol abuse in the past year are excluded.
Patients who have had a major surgery within the last 3 months cannot participate.

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Azienda Ospedaliera Universitaria Citta' Della Salute E Della Scienza Di Torino
Verified
Turin, Italy
Hospital Sant Joan De Deu Barcelona
Verified
Esplugues De Llobregat, Spain
Hospital Vall d’Hebron
Verified
Barcelona, Spain

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Recruiting

Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Ravulizumab is a medication used in this clinical trial to help treat children with a condition called Primary Immunoglobulin A Nephropathy (IgAN). This condition affects the kidneys, and the medication works by blocking a part of the immune system that can cause damage to the kidneys. In this study, the medication is given through an intravenous (IV) infusion, which means it is delivered directly into the bloodstream through a vein. The goal of using this medication in the trial is to understand how it behaves in the body, how it affects the disease, and to ensure it is safe and effective for children, based on what is already known from studies in adults.

Ravulizumab

What is already known about the treatment

Ravulizumab – Ravulizumab is administered through an intravenous (IV) infusion, meaning it is delivered directly into the bloodstream through a vein. It is currently being studied in clinical trials for its use in treating children with Immunoglobulin A Nephropathy (IgAN), a kidney disease. The medication works by blocking a part of the immune system that can cause inflammation and damage to the kidneys. Ravulizumab is classified as a monoclonal antibody, which is a type of drug designed to target specific proteins in the body.

Investigated diseases

Immunoglobulin A Nephropathy – Immunoglobulin A Nephropathy, also known as IgA Nephropathy, is a kidney disease that occurs when the antibody immunoglobulin A (IgA) builds up in the kidneys. This buildup causes inflammation that can affect the kidneys' ability to filter waste from the blood. The disease often progresses slowly over many years. In the early stages, it may not cause any symptoms, but as it advances, it can lead to blood in the urine, swelling in the hands and feet, and high blood pressure. Over time, the persistent inflammation can lead to scarring of the kidneys, known as fibrosis. This scarring can eventually impair kidney function significantly.