RecruitingRare disease

Study of Danicopan with Ravulizumab or Eculizumab for Children with Paroxysmal Nocturnal Hemoglobinuria and Extravascular Hemolysis

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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 rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is testing a treatment involving a medication called Danicopan, which is used in combination with other treatments like Ravulizumab or Eculizumab. These medications are designed to help manage the symptoms of PNH, particularly in children who experience significant breakdown of red blood cells outside of blood vessels, known as extravascular hemolysis.

The purpose of the study is to evaluate how effective Danicopan is when added to the existing treatments of Ravulizumab or Eculizumab. Participants in the study will take Danicopan in the form of a film-coated tablet, which is taken orally. The study will monitor changes in the participants' health over a period of time, specifically looking at improvements in their blood levels and overall well-being. The study will last for several weeks, and participants will have regular check-ups to assess their progress.

This trial is open-label, meaning both the participants and the researchers know which treatment is being administered. The study aims to provide valuable information on how well Danicopan works in helping children with PNH manage their condition more effectively. Participants will be closely monitored to ensure their safety and to gather data on the treatment's impact on their health.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment phase
The study involves the use of danicopan as an additional treatment to either ravulizumab or eculizumab. These medications are used to treat a condition called paroxysmal nocturnal hemoglobinuria (PNH), which affects the blood.
Participants will take danicopan in the form of a film-coated tablet by mouth. The exact dosage and frequency will be determined by the study team based on individual needs and responses.
The primary goal during this phase is to assess the change in hemoglobin levels from the start of the study to week 12.
Step 2
Ongoing treatment and monitoring
Participants will continue to receive ravulizumab or eculizumab as part of their regular treatment regimen. The dosage of these medications should remain stable during the first 12 weeks of the study.
Regular monitoring will occur to assess the effectiveness of the treatment, including changes in hemoglobin levels and other blood parameters.
Participants will be evaluated for any side effects or adverse events throughout the study period.
Step 3
Evaluation and follow-up
At weeks 12 and 24, participants will undergo assessments to evaluate the overall impact of the treatment on their condition.
These assessments will include measuring changes in hemoglobin levels, reticulocyte counts, and quality of life scores.
The study will also monitor the need for blood transfusions and any changes in fatigue levels.
Step 4
Completion of study participation
The study is expected to continue until February 2028, with recruitment starting in January 2025.
Participants will complete their involvement in the study after the final assessments are conducted and all data is collected.
The results will contribute to understanding the effectiveness and safety of danicopan as an add-on treatment for PNH.

Who can join the trial?

12 criteria

Participants must be between 12 and less than 18 years old at the time of signing the informed consent.
Participants must have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood condition.
Participants must have CS-EVH, which means they have anemia with hemoglobin (Hgb) levels of 10.5 g/dL or lower and a high reticulocyte count (a type of blood cell) of 100 x 10⁹/L or more.
Participants must have been treated with either ravulizumab or eculizumab for at least 12 weeks before starting the study, with a stable dose during this time.
Participants must weigh more than 25 kg (about 55 pounds).
Participants must be vaccinated against meningococcal infection (a type of bacterial infection) from specific serogroups within 3 years before or at least 14 days before starting the study. If not, they will need to be vaccinated and may receive antibiotics for at least 2 weeks after vaccination.
Participants must be vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae according to local guidelines.
Participants can be male or female.
Female participants must not be pregnant or breastfeeding. If they are of childbearing potential, they must use an effective method of contraception during the study and have a negative pregnancy test before starting.
The participant's legal guardian or legally authorized representative must provide written informed consent, and the participant must provide assent if applicable.
A legal guardian or primary caregiver must be available to assist with study visits, follow-up, and maintaining health records.
Participants must have access to emergency medical care.

Who cannot join the trial?

10 criteria

Patients who have any other serious health condition that might interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who have a known allergy or adverse reaction to the study medications.
Patients who are unable to comply with the study procedures or follow-up visits.
Patients who are pregnant or breastfeeding.
Patients who have a history of drug or alcohol abuse.
Patients who have an infection that requires treatment with antibiotics.
Patients who have a history of cancer, except for certain types of skin cancer.
Patients who have a condition that affects their immune system, making them more prone to infections.

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Verified sites

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Robert Debre University Hospital
Verified
Paris, France

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Investigated drugs

Danicopan
is being studied as an additional treatment for children with a condition called Paroxysmal Nocturnal Hemoglobinuria (PNH), which causes the destruction of red blood cells. This medication is being tested to see if it can help improve blood levels when used alongside other treatments.
Ravulizumab
is a medication used to treat PNH by preventing the breakdown of red blood cells. It works by blocking a part of the immune system that attacks these cells, helping to reduce symptoms and improve quality of life.
Eculizumab
is another treatment for PNH that also helps prevent the destruction of red blood cells. Like ravulizumab, it targets the immune system to protect red blood cells from being destroyed, which can help manage the symptoms of the disease.

Danicopan

What is already known about the treatment

Danicopan
Danicopan is administered orally and is currently being studied in clinical trials as an add-on treatment for pediatric patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who experience significant extravascular hemolysis. It is not yet widely used in standard medical practice, as it is still under investigation. The main therapeutic indication is to improve hemoglobin levels in these patients. Danicopan works by inhibiting a specific enzyme in the complement pathway, which helps reduce the breakdown of red blood cells. It is classified as a complement inhibitor.
Ravulizumab
Ravulizumab is administered through intravenous infusion and is an established treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). It is approved for use in medical practice and is well-documented in medical literature. The primary therapeutic indication is to prevent the destruction of red blood cells in PNH patients. Ravulizumab works by blocking a part of the immune system called the complement system, which is responsible for the breakdown of red blood cells. It is classified as a complement inhibitor.
Eculizumab
Eculizumab is given through intravenous infusion and is a well-known treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). It is widely used in clinical settings and is supported by extensive medical literature. The main therapeutic indication is to reduce hemolysis and improve symptoms in PNH patients. Eculizumab functions by inhibiting the complement system, preventing the immune-mediated destruction of red blood cells. It is also classified as a complement inhibitor.

Investigated diseases

Paroxysmal Nocturnal Hemoglobinuria – This is a rare blood disorder where red blood cells break down earlier than normal. It is caused by a mutation in the PIGA gene, leading to a deficiency of certain proteins that protect red blood cells from the immune system. As a result, red blood cells are destroyed, leading to symptoms such as fatigue, shortness of breath, and dark-colored urine, especially in the morning. The disease can also cause blood clots and low levels of red blood cells, known as anemia. Over time, the destruction of red blood cells can lead to complications affecting various organs. The condition is chronic and can vary in severity among individuals.