Ravulizumab for Thrombotic Microangiopathy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety and effectiveness of a treatment called ravulizumab (also known as Ultomiris) for thrombotic microangiopathy (TMA), a serious condition that can occur after a bone marrow transplant. The treatment is administered through an IV over 26 weeks, followed by another 26 weeks of check-ups. Children and teenagers who underwent a bone marrow transplant in the past year and still exhibit TMA symptoms might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants an opportunity to contribute to potentially groundbreaking treatment advancements.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that ravulizumab is likely to be safe for humans?
Research has shown that ravulizumab, a treatment under study for thrombotic microangiopathy (TMA), has been safe in other applications. In studies involving adults and children with atypical Hemolytic Uremic Syndrome (aHUS), ravulizumab was generally well-tolerated.
Some participants experienced side effects after starting treatment, but these were mostly manageable, with serious side effects being less common. Ravulizumab is already approved for treating aHUS, indicating its safety is well-established for that condition.
In another study on TMA in adults, the safety profile of ravulizumab was similar to that observed in aHUS, with no new safety concerns. While side effects can occur, they are usually not severe.
Based on past studies, ravulizumab appears to be a treatment that people can generally tolerate well.12345Why do researchers think this study treatment might be promising?
Ravulizumab is unique because it targets and inhibits a specific part of the immune system called the complement pathway, which plays a crucial role in thrombotic microangiopathy (TMA). Unlike standard treatments that might focus on managing symptoms or complications, Ravulizumab directly addresses the underlying cause of the disease. This targeted approach not only has the potential to reduce the frequency and severity of TMA episodes but also could lead to longer-lasting protection with fewer doses, making it a more convenient option for patients. Researchers are excited because this could represent a significant advancement in both effectiveness and quality of life for those affected by TMA.
What evidence suggests that ravulizumab might be an effective treatment for HSCT-TMA?
Research has shown that ravulizumab, the treatment under study in this trial, yields promising results for treating thrombotic microangiopathy (TMA). In children who underwent a stem cell transplant, 87% of those treated with ravulizumab survived for at least 26 weeks. This treatment blocks part of the immune system that can cause harm, helping to prevent problems with blood vessels. Ravulizumab has already proven effective for atypical hemolytic uremic syndrome, a rare condition that causes blood clots, in both adults and children. These findings suggest it could be a helpful option for TMA after transplants, a condition with few treatment options.12456
Are You a Good Fit for This Trial?
This trial is for children and teens from 1 month to under 18 years who've had a stem cell transplant in the last year and are suffering from HSCT-TMA. They must be able to consent, use effective birth control if applicable, be vaccinated against meningococcal infections or on antibiotics, weigh at least 5 kg, and not have certain other health conditions.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ravulizumab plus Best Supportive Care for 26 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Ravulizumab
Ravulizumab is already approved in United States, European Union for the following indications:
- Paroxysmal nocturnal hemoglobinuria (PNH)
- Atypical hemolytic uremic syndrome (aHUS)
- Paroxysmal nocturnal haemoglobinuria (PNH)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion
Lead Sponsor
Marc Dunoyer
Alexion
Chief Executive Officer since 2021
PhD in Molecular Biology, University of Brussels
Christophe Hotermans
Alexion
Chief Medical Officer since 2021
MD, University of Leuven
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology
Alexion Pharmaceuticals
Lead Sponsor
Marc Dunoyer
Alexion Pharmaceuticals
Chief Executive Officer since 2021
B.A. in Psychology from the University of New Hampshire
Christophe Hotermans
Alexion Pharmaceuticals
Chief Medical Officer since 2023
MD