A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
This study, called "A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients," is for people living with Fabry disease. It aims to understand how well a treatment called Elfabrio® (peguni-gal-sidase-alfa) works and if it's safe when used in everyday healthcare settings across different countries. Researchers will look at existing health information and collect new data from patients receiving Elfabrio®. This is not a study where you try a new medicine; instead, it observes people who are already on or are about to start Elfabrio® as part of their regular care. The goal is to gather important information about this treatment to help doctors and patients in the future.
At a glance
What is this study about?
This study is about a medication called Elfabrio® (which contains peguni-gal-sidase-alfa), used to treat Fabry disease. Fabry disease is a rare genetic condition that can affect many parts of the body, including the heart, kidneys, and nervous system.
Researchers want to see how Elfabrio® works in real life. This means they will be observing people who are already taking or are planning to start taking Elfabrio® as part of their regular treatment. Think of it like watching how a car performs on different roads after it's been bought, rather than testing it on a closed track. They'll collect information from your medical records and ask you some questions about your health and how you feel. Doctors call this an 'observational' study because they are mainly observing what happens during your usual care, not giving you new or different treatments.
This study is important because it helps us understand the long-term effects of Elfabrio® in a wide variety of patients. By gathering this information, doctors and scientists can learn more about how the medicine helps people with Fabry disease and can improve patient care in the future. It’s also interesting because it combines looking back at old medical records with collecting new information as time goes on, giving a more complete picture.
Key takeaways
- It's an observational study for people with Fabry disease on Elfabrio®.
- It aims to understand how Elfabrio® works in real-world settings.
- You'll receive your usual medical care and treatment.
- Data will be collected from your medical records and questionnaires.
- Joining helps doctors learn more about Fabry disease treatment.
- You can withdraw from the study at any time without affecting your care.
Who may be eligible?
To join this study, you need to be an adult, aged 18 or older, and have a confirmed diagnosis of Fabry disease through genetic testing. You should either be already taking Elfabrio® or be planning to start it as part of your treatment plan. It's also important that you don't have any medical reasons that would prevent you from having a special heart scan called a cardiac MRI.
There are a few different groups within the study. Some people will be included if their Fabry disease has affected their heart, and they might need special heart scans. Others might be eligible if they haven't had much treatment before for their kidney function, or if they've participated in a previous study about this medication. Don't worry if those details sound a bit complex; your doctor will help figure out which group, if any, you fit into.
You cannot join the study if you are pregnant when you agree to take part, or if you have any medical condition that makes it unsafe for you to have an MRI scan, especially if you have had a bad reaction to the contrast dye used in these scans in the past. Your doctor will discuss all these points with you to make sure the study is a good fit.
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.
- Are you 18 years old or older?
- Do you have a genetically confirmed diagnosis of Fabry disease?
- Are you currently taking Elfabrio® or planning to start it soon?
- Can you have a type of heart scan called a cardiac MRI?
- Are you able to fill out online health questionnaires?
- Are you not pregnant at this time?
What does participation involve?
If you decide to take part, you won't be given any new or experimental treatments. Instead, the study will gather information about your health and your treatment with Elfabrio® as you receive your usual care from your doctor. This might involve allowing the researchers to look at your past and future medical records, including results from blood tests and heart scans (like cardiac MRI) that you would already be having as part of your Fabry disease management. You'll also be asked to complete some questionnaires, which you can do electronically, to share how you are feeling and how the treatment is affecting you. There's no specific length mentioned for how long you'd be involved, but observational studies often track patients over many months or even years as they continue their ongoing treatment.
Potential risks and benefits
Locations (10)
- University of Alabama at BirminghamVerified postcodeBirmingham, United States· Recruiting
- Emory University School of MedicineVerified postcodeAtlanta, United States· Recruiting
- Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of ChicagoVerified postcodeChicago, United States· Recruiting
- University of Iowa Hospitals and ClinicsVerified postcodeIowa City, United States· Recruiting
- Infusion AssociatesVerified postcodeGrand Rapids, United States· Recruiting
- Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)Verified postcodeFairfax, United States· Recruiting
- General Hospital Slovenj GradecVerified postcodeSlovenj Gradec, Slovenia· Recruiting
- University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth HospitalVerified postcodeEdgbaston, United Kingdom· Recruiting
- Salford RoyalVerified postcodeSalford, United Kingdom· Recruiting
- The Royal Free HospitalVerified postcodeLondon, United Kingdom· Recruiting
Common questions
What is Fabry disease?
Fabry disease is a rare genetic condition that happens when a certain enzyme is missing or doesn't work correctly, leading to a build-up of fatty substances in the body's cells, which can affect many organs.
What is Elfabrio®?
Elfabrio® is a medicine used to treat Fabry disease. It works by replacing the missing or faulty enzyme in your body.
Will I get different treatment if I join this study?
No, if you join this study, you will continue to receive the same Elfabrio® treatment and medical care that your doctor has already prescribed for you. The study simply observes your progress.
Do I have to have an MRI scan for this study?
You might need to have an MRI scan if it is already part of your routine care for monitoring your Fabry disease, especially if your heart is affected. The study doesn't ask you to have scans you wouldn't normally have.
Can I leave the study if I change my mind?
Yes, you are free to leave the study at any time and for any reason. Your decision will not affect your medical care or your relationship with your healthcare team.
How to find out more
Medical Information Chiesi
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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