Registry for Atypical Hemolytic Uremic Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gather safety information on two treatments, eculizumab and ravulizumab, for people with atypical Hemolytic-Uremic Syndrome (aHUS), a rare disease affecting the blood and kidneys. It focuses on patients either already receiving these treatments or not yet started on them. Suitable candidates include those diagnosed with aHUS, with or without a known genetic issue related to the disease.
As a Phase 4 trial, this research helps to understand how these FDA-approved treatments benefit more patients. It offers an opportunity to contribute to broader knowledge and potentially improve care for others with aHUS.
Do I have to stop taking 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.
What is the safety track record for eculizumab and ravulizumab?
A previous study found that eculizumab generally carries a low risk of harmful effects. Most safety information comes from clinical trials, and no major side effects are expected with correct use. However, limited information exists about its safety in everyday use. Research shows that ravulizumab is effective but can cause common side effects like diarrhea and cold-like symptoms. There is also a risk of serious infections from Neisseria meningitidis, making vaccination important for those using this treatment. Overall, both treatments are considered relatively safe, but some risks require management.12345
Why are researchers enthusiastic about this study treatment?
Researchers are excited about Eculizumab and Ravulizumab for treating atypical Hemolytic-Uremic Syndrome (aHUS) because of their unique mechanisms of action. Both drugs are monoclonal antibodies that target the complement protein C5, effectively preventing the overactive immune response that characterizes aHUS. What sets Ravulizumab apart is its longer duration of action, meaning it requires less frequent dosing compared to Eculizumab, making it more convenient for patients. This extended dosing schedule could lead to better adherence and improved quality of life for those managing this rare condition.
What is the effectiveness track record for eculizumab and ravulizumab?
Research has shown that eculizumab can extend patients' lives and reduce the risk of blood clots. It improves survival rates by 49% and lowers the risk of blood clots by about 60%. Additionally, it alleviates symptoms in conditions like myasthenia gravis, resulting in stronger muscles and an enhanced quality of life.
For ravulizumab, studies indicate it consistently controls symptoms in conditions like generalized myasthenia gravis. It remains safe and effective over the long term, making it a reliable option for symptom management. Both treatments target the immune system to protect blood vessels and organs from damage.678910Who Is on the Research Team?
Nuria Saval
Principal Investigator
Alexion Pharmaceuticals, Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive treatment with eculizumab or ravulizumab, or other disease management approaches
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term follow-up
Collection and evaluation of long-term safety and effectiveness data
What Are the Treatments Tested in This Trial?
Interventions
- Eculizumab
- Ravulizumab
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
Syneos Health
Collaborator
Citations
Data from the International PNH Registry
We demonstrate that treatment with eculizumab conferred a 49% relative benefit in survival and an approximately 60% reduction in TE/MAVE risk.
Real-World Eculizumab Effectiveness on US Patient ...
Results from this study suggest that treatment with eculizumab reduced patients' treatment burden (shown by the decrease in concomitant prednisone use) and ...
Long‐term effectiveness of eculizumab: Data from the ...
Long-term effectiveness data of eculizumab on patient survival and prevention of clinical outcomes such as TEs have been previously ...
The effectiveness and value of eculizumab ...
At week 26, the proportion of patients with minimal symptom expression (MG-ADL score of 0 or 1) was much greater in the eculizumab group (21.4% vs 1.7%, P = ...
First analysis of the Myasthenia Gravis SPOTLIGHT Registry
Clinical trials have demonstrated improved functional ability, muscle strength, and quality of life (QOL) in patients treated with eculizumab or ...
Safety Data Sheet
Wear appropriate protective eyeglasses or chemical safety goggles as described by OSHA. Body Protection. Wear compatible chemical-resistant ...
7.
file.medchemexpress.com
file.medchemexpress.com/batch_PDF/HY-P9914/Eculizumab-SDS-MedChemExpress.pdfSafety Data Sheet
Eculizumab. Catalog No. : HY-P9914. CAS No. : 219685-50-4. 1.2 Relevant identified uses of the substance or mixture and uses advised against.
8.
abmole.com
abmole.com/literature/eculizumab-msds.html?srsltid=AfmBOorLumPeXGxzp5792aQyqpw-_oEgaGb4o4JwmQamCSndphnaW-_MMaterial Safety Data Sheet of Eculizumab
Avoid inhalation, contact with eyes and skin. Avoid dust and aerosol formation. Use only in areas with appropriate exhaust ventilation. 7.2, Conditions for safe ...
Real-world safety profile of eculizumab: an analysis of FDA ...
Most of the safety data regarding eculizumab came from clinical trials, while its safety information in the real world is still limited.
SAFETY DATA SHEET
Based on composition, the mixture has low acute toxicity and no adverse effects for human health are expected under applicable conditions of ...
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