A Phase 3, Open-Label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Activity, and Safety of Ravulizumab Administered Subcutaneously in Pediatric Participants (2 to < 18 years of age) with Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS)
This study is testing a medicine called ravulizumab, also known as Ultomiris, in children aged 2 to 18 who have either Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS). These are both rare blood conditions. The main goal is to understand how the medicine moves through the body (pharmacokinetics) and how it affects the body (pharmacodynamics) when given as an injection under the skin. We also want to check how active the medicine is and if it’s safe for children. This is a "Phase 3" study, which means it's one of the final steps before a new medicine might be approved for wider use.
At a glance
What is this study about?
This research study is about helping children and teenagers between 2 and 18 years old who have one of two rare blood conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS). In these conditions, the body's immune system can mistakenly attack healthy blood cells, leading to serious health problems.
The study is looking at a medicine called ravulizumab, which is also known as Ultomiris. Currently, this medicine is often given through a drip into a vein (infusion). This study wants to see if giving the medicine as an injection under the skin (subcutaneously) is a good alternative. This could make it much easier for patients and their families, as they might be able to administer the medicine at home instead of needing to go to a clinic for infusions.
We will be carefully tracking several things to see how well the medicine works and if it's safe. This includes measuring how much medicine is in the blood, how it affects certain blood markers related to the diseases, and how patients feel in their daily lives. For children with PNH, we'll check things like how tired they are and if they need fewer blood transfusions. For those with aHUS, we'll look at kidney function. The overall aim is to find a more convenient and effective way to manage these challenging conditions.
Key takeaways
- This study is for children (2-18 years) with rare blood conditions PNH or aHUS.
- It tests a new way to give the medicine ravulizumab (Ultomiris) – by injection under the skin.
- The study will check how safe and effective the injection method is.
- It aims to improve treatment convenience and quality of life for children.
- Participation involves regular clinic visits for about a year to monitor health and treatment effects.
Who may be eligible?
This study is specifically looking for children and teenagers between 2 and 18 years old. They must have been diagnosed with either Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS).
There will be other specific medical requirements that your doctor will need to check to make sure the study is a safe and suitable option for your child. These will include factors like their general health, other medical conditions they might have, and any other medicines they are taking.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child between 2 and 18 years old?
- Has your child been diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH)?
- Or has your child been diagnosed with Atypical Hemolytic Uremic Syndrome (aHUS)?
- Is your child generally able to attend regular clinic appointments for about a year?
What does participation involve?
If your child takes part in this study, they will receive the study medicine, ravulizumab, which will be given as an injection under the skin. The study will last for about a year. During this time, there will be several visits to the clinic where doctors and nurses will take blood samples to measure how the medicine is working and how it affects their body.
They will also monitor your child's general health, check for any side effects, and ask about their quality of life, including how tired they feel. For children with PNH, they will check things like their need for blood transfusions. For those with aHUS, kidney function will be monitored carefully. There will also be checks on the injection device to make sure it's working correctly and that the full dose is given. You can withdraw your child from the study at any time.
Potential risks and benefits
Locations (2)
- —UnverifiedItaly
- —UnverifiedSpain
Common questions
What is PNH or aHUS?
PNH (Paroxysmal Nocturnal Hemoglobinuria) and aHUS (Atypical Hemolytic Uremic Syndrome) are both rare conditions where the body's immune system mistakenly attacks its own healthy blood cells, leading to serious health issues.
What is ravulizumab (Ultomiris)?
Ravulizumab (Ultomiris) is a medicine used to treat PNH and aHUS. This study is looking at a new way to give it via an injection under the skin, rather than through a drip in the arm.
Why is this study important for children?
This study is important because it specifically focuses on children and teenagers, trying to find a more comfortable and easier way for them to receive their vital medication, which could improve their daily lives.
Will my child have to travel a lot for the study?
The study involves regular visits to a clinic for blood tests and health checks. The research team will discuss the frequency and location of these visits with you.
What if we change our minds about participating?
You and your child have the right to leave the study at any time, for any reason, without it affecting your child's medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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