ATLAS-PEDS: An open-label, multinational study of fitusiran prophylaxis in pediatric subjects ages 1 to less than 12 years with hemophilia A or B
Haemophilia is a rare condition where blood does not clot as well as it should, leading to easy bruising and painful bleeding inside joints or muscles. The ATLAS-PEDS clinical trial is looking at a new preventative treatment called fitusiran (SAR439774) for boys aged between 1 and 11 years old who have haemophilia A or B. Fitusiran works differently from traditional replacement clotting factors. It reduces levels of a natural protein called antithrombin, which allows the body to form blood clots more readily. In this study, every child receives fitusiran—there is no dummy drug (placebo). Doctors will carefully monitor the children's blood clotting levels, check how fitusiran moves through their bodies, and record any side effects. The goal is to discover if fitusiran is safe, well-tolerated, and effective at preventing bleeds in children.
At a glance
What is this study about?
Haemophilia is a lifelong condition where the body lacks enough clotting factors, which are special proteins needed to stop bleeding. Without these proteins, even minor injuries can cause prolonged bleeding, and spontaneous bleeding into joints or muscles can cause pain and damage. Most current treatments involve regular injections into a vein to replace the missing clotting proteins, which can be difficult for young children and their families.
This study, called ATLAS-PEDS, is testing a new approach using a medicine called fitusiran. Instead of replacing the missing clotting factor, fitusiran reduces the amount of antithrombin, an opposing protein that naturally prevents clots from forming. By lowering antithrombin, the blood can clot more easily, helping to restore a healthy clotting balance and stop bleeds before they start.
ATLAS-PEDS is an 'open-label' study, meaning parents, doctors, and researchers will all know that the child is receiving fitusiran. Researchers are measuring how fitusiran affects antithrombin levels and how safe it is for young children. This research matters because finding simpler, longer-lasting ways to prevent bleeds could greatly improve the daily lives and long-term joint health of young children growing up with haemophilia.
Key takeaways
- Testing a preventative medicine called fitusiran for boys aged 1 to 11 with haemophilia A or B.
- Fitusiran works by lowering antithrombin to help blood clot more easily.
- All children in this study receive the active medicine; there is no placebo.
- The medicine is given as an injection under the skin rather than into a vein.
- Taking part is entirely voluntary, and you can leave the study at any time.
Who may be eligible?
This trial is designed specifically for young boys aged from 1 year old up to 11 years old who have been diagnosed with either haemophilia A or haemophilia B. Because haemophilia is a genetic condition that almost exclusively affects males, the study is open to male participants only.
Children may not be able to join if they have other serious health problems, such as severe liver or kidney conditions, or if they are taking specific medications that could interfere with the study medicine. A full medical check will be carried out by the study doctors before any child can take part.
Your child's haemophilia care team can look at their medical history, their current treatment routine, and their overall health to help you decide if this study might be suitable.
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.
- My child is male.
- My child is aged between 1 and 11 years old.
- My child has been diagnosed with haemophilia A or haemophilia B.
- We are able to attend regular hospital appointments for check-ups and blood tests.
What does participation involve?
Taking part involves regular visits to a specialist hospital or clinical trial centre. Your child will receive fitusiran, which is usually given as an injection just under the skin. During visits, the medical team will carry out health checks, take blood samples to measure clotting levels and drug levels, and ask about any bleeds or symptoms your child has experienced. Parents may be asked to keep a simple diary of any bleeding episodes or side effects. The study lasts over several months to monitor your child's health closely, and regular follow-up visits will be scheduled throughout.
Potential risks and benefits
Locations (2)
- —UnverifiedItaly
- —UnverifiedSpain
Common questions
Will my child receive a dummy treatment (placebo)?
No. This is an open-label trial, which means all children who take part will receive the active study medicine, fitusiran.
How is the study medicine given?
Fitusiran is given as an injection under the skin (subcutaneously), rather than directly into a vein.
What happens if my child has a bleed during the study?
The study team will provide a clear, tailored plan on how to treat sudden bleeds safely using your child's usual emergency clotting treatments.
Do we have to pay to take part?
No, the study medication and trial-related tests are free. Clinical trials often help cover reasonable travel and parking costs for hospital visits.
Can we change our minds after starting?
Yes. You can choose to stop your child's participation at any point without giving a reason, and your normal healthcare team will continue their care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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