Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome

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What is this study about?

This clinical trial is focused on studying two rare blood disorders: Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS). These conditions can cause severe health issues, including the destruction of red blood cells, blood clots, and kidney problems. The study will use a treatment called Ravulizumab, which is a type of medication known as a monoclonal antibody. This medication is designed to help manage these disorders by targeting specific proteins in the body that contribute to the symptoms.

The purpose of the study is to understand how Ravulizumab works in children aged 2 to under 18 years who have either PNH or aHUS. Participants will receive the medication through an injection under the skin. The study will last for about a year, during which time the effects of the medication will be closely monitored. This includes looking at how the body processes the drug and how it affects the symptoms of the diseases. The study will also assess the safety of the medication and any side effects that may occur.

Throughout the study, participants will have regular check-ups to monitor their health and the effectiveness of the treatment. The study aims to provide valuable information that could improve the management of PNH and aHUS in children. By participating, researchers hope to gather data that could lead to better treatment options for these serious conditions. The study will also evaluate the use of a special device that helps deliver the medication, ensuring it is administered correctly and safely.

1 joining the study

Upon joining the study, participants will be informed about the trial’s purpose, procedures, and potential risks and benefits. Consent will be obtained from the participant’s legal guardian or representative, and assent from the participant if applicable.

2 initial assessment

Participants will undergo an initial assessment to confirm eligibility. This includes a review of medical history and a series of tests to ensure the participant meets the study criteria.

3 treatment administration

Participants will receive the medication ravulizumab through a subcutaneous injection. The dosage and frequency will be determined by the study protocol and adjusted based on the participant’s response and condition.

4 regular monitoring

Participants will attend regular visits to monitor their health and response to the treatment. This includes blood tests and other assessments to track the medication’s effects and any side effects.

5 follow-up assessments

Throughout the study, participants will undergo follow-up assessments to evaluate the treatment’s effectiveness and safety. These assessments will occur at specified intervals, such as Day 1, Day 15, and Day 71, and continue through Week 52.

6 completion of the study

At the end of the study period, participants will have a final assessment to gather comprehensive data on their health status and the treatment’s impact. This information will contribute to the study’s overall findings.

Who Can Join the Study?

Must be between 2 and 17 years old at the time of giving consent.
Can be either male or female.
Must weigh at least 10 kg (about 22 pounds) at the time of screening.
Must have a legal guardian or representative who can give written consent, and the participant must be able to give written assent if required.
Must be vaccinated against meningococcal infection (a type of bacterial infection) from specific groups (A, C, Y, W135, and B) within the last 3 years or at least 2 weeks before starting the study. If not, they must receive the vaccine and take preventive antibiotics for at least 2 weeks if the study starts less than 2 weeks after vaccination.
Must have received vaccination for Streptococcus pneumoniae (a type of bacteria that can cause infections) according to local guidelines.
Must have received vaccination for Haemophilus influenzae type b (a type of bacteria that can cause infections) according to local guidelines.
For participants with Paroxysmal Nocturnal Hemoglobinuria (PNH):
- Must have a confirmed diagnosis of PNH through specific blood tests.
- If not previously treated with complement inhibitors, must have shown symptoms related to PNH in the last 3 months, such as fatigue, dark urine, abdominal pain, shortness of breath, anemia, history of blood clots, difficulty swallowing, or erectile dysfunction.
- Must meet specific laboratory test criteria for LDH (a blood test) based on treatment history.
For participants with Atypical Hemolytic Uremic Syndrome (aHUS):
- If not previously treated with complement inhibitors, must show signs of Thrombotic Microangiopathy (TMA), which includes low platelet count, evidence of red blood cell breakdown, and kidney injury based on specific laboratory tests.
- If previously treated with eculizumab or ravulizumab, must have a confirmed diagnosis of aHUS with specific laboratory findings at the time of the TMA event.
- Must show clinical evidence of response to previous treatment with stable TMA parameters at screening.
- For those with a kidney transplant, must have a known history of aHUS or persistent evidence of TMA after changing certain medications.
- For those with TMA onset after childbirth, must have persistent evidence of TMA for more than 3 days after delivery.
Participants who have been treated with eculizumab or ravulizumab must have been on the treatment for at least 90 days before screening, with no missed doses in the last 2 months and no more than 2 doses outside the scheduled time.
Female participants who can have children and male participants must agree to follow specific contraception guidelines during the study.

Who Cannot Join the Study?

Patients who are not diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS) cannot participate.
Patients who are not within the specified age range for the study cannot participate.
Patients who are not able to follow the study procedures or comply with the study requirements cannot participate.
Patients who have any other medical conditions that might interfere with the study cannot participate.
Patients who are currently participating in another clinical trial cannot participate.
Patients who have a history of allergic reactions to the study medication cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have received certain medications recently that might interfere with the study cannot participate.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Universidade De Santiago De Compostela	Santiago De Compostela	Spain
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Auiqiki Oobqflbykcr Utyvecrqlwkqz Cpgrfmjzfpzv Ddish Sybtwm E Dhana Sxvwxvh Dd Tjfsxj	Turin	Italy
Hpxhxywk Vuij dkoubvsp	Barcelona	Spain
Hrmhnyda Ucvhnfussgtte ds A Cnryko	A Coruna Galicia	Spain

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	30.08.2024
Spain	Not recruiting	30.08.2024

Trial locations

Investigated drugs:

Ravulizumab

Ravulizumab is a medication used in this clinical trial to help treat children with certain blood disorders, specifically Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS). These conditions can cause problems with blood cells and the kidneys. Ravulizumab works by blocking a part of the immune system that can attack the body’s own cells, which helps to prevent the destruction of red blood cells and reduces the risk of kidney damage. In this study, the medication is given as an injection under the skin, which is known as subcutaneous administration. The goal is to understand how the medication moves through the body, how it affects the body, and to ensure it is safe and effective for children with these conditions.

Paroxysmal Nocturnal Hemoglobinuria – Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare blood disorder characterized by the destruction of red blood cells. This destruction occurs due to a defect in the surface proteins that normally protect these cells from the immune system. As the disease progresses, patients may experience symptoms such as fatigue, shortness of breath, and dark-colored urine, especially in the morning. Over time, the breakdown of red blood cells can lead to anemia and other complications. The disease can also cause blood clots, which may lead to further health issues. PNH is a chronic condition that requires ongoing monitoring and management.

Atypical Hemolytic Uremic Syndrome – Atypical Hemolytic Uremic Syndrome (aHUS) 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. The disease progresses as the small blood vessels in the kidneys become damaged, leading to reduced kidney function. Patients may experience symptoms such as fatigue, high blood pressure, and decreased urine output. As aHUS advances, it can lead to serious complications affecting multiple organs. The condition is chronic and requires careful management to prevent further damage.

Trial ID:

2022-502335-19-00

Protocol code:

ALXN1210-PED-316

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome

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