Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
This study, called the Maternal and Postnatal Outcomes Study (MOS), is looking into the safety of a medicine called Elfabrio for women with Fabry disease. It focuses on women who have used Elfabrio within 30 days before pregnancy or during pregnancy and breastfeeding. Researchers want to see how these pregnancies turn out for both the mothers and their babies. They'll be checking for any health problems in the mothers and any birth defects or other issues in the newborns. It's an observation study, meaning no new treatments are given; it just collects information over 10 years to understand the real-world effects.
At a glance
What is this study about?
If you are a woman with Fabry disease and are pregnant or thinking about getting pregnant, this study might be of interest to you. It's called the Maternal and Postnatal Outcomes Study (MOS) and it aims to understand how safe a medicine called Elfabrio is for expectant mothers and their babies. Doctors want to learn if using Elfabrio shortly before or during pregnancy, or while breastfeeding, has any effects on the mother's health, the pregnancy itself, or the baby's development.
This study is important because it will help doctors and patients make better decisions about treatment for Fabry disease during such a crucial time. It's not a study where you'll be given a new drug or told to change your current treatment. Instead, it's an 'observational' study. This means it simply collects information about what naturally happens to women with Fabry disease who are using or have used Elfabrio around the time of pregnancy and breastfeeding.
The study will follow women and their babies for a long time – up to 12 months after the baby is born. Researchers will look at things like how healthy the mother is, any problems during pregnancy, how the birth goes, and the baby's health in their first year, including checking for any birth defects.
Key takeaways
- This study focuses on women with Fabry disease taking Elfabrio during pregnancy or breastfeeding.
- It aims to understand the safety and outcomes for both mothers and their babies.
- It is an 'observational' study, meaning no changes to your current treatment.
- Information will be collected securely online for up to 12 months after birth.
- Insights gained will help improve care for future patients.
- You can withdraw at any time without affecting your medical care.
Who may be eligible?
You might be able to take part in this study if you are a woman with Fabry disease and have received the medicine Elfabrio. This includes if you had Elfabrio within 30 days before getting pregnant, or at any point during your pregnancy or while you were breastfeeding.
Your baby would also be part of the study. There are no other health reasons that would stop you from joining this study. You (or your parent/guardian if you cannot legally consent yourself) would need to be able to understand and sign a consent form.
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.
- I am a woman with Fabry disease.
- I have taken Elfabrio within 30 days before conception, or during pregnancy, or while breastfeeding.
- I (or my legal guardian) can understand and sign a consent form.
- My baby is also part of the study.
- I am comfortable with my medical information being collected for research.
What does participation involve?
If you decide to take part, you or your doctor will share information about your health and your pregnancy. This information will be put into a secure online system. This can be done either during your pregnancy or after your baby is born. The researchers will collect details about your pregnancy and your baby's health from the time you enrol until your baby is 12 months old. This study is observational, meaning it won't change any of your current medical care or treatment decisions; doctors will continue to treat you as they normally would.
Potential risks and benefits
Locations (5)
- No physical study sites - Decentralized, web-based registryVerified postcodeWashington D.C., United States· Recruiting
- No physical study sites - Decentralized, web-based registryVerified postcodeBerlin, Germany· Recruiting
- No physical study sites - Decentralized, web-based registryVerified postcodeRome, Italy· Recruiting
- No physical study sites - Decentralized, web-based registryVerified postcodeMadrid, Spain· Recruiting
- No physical study sites - Decentralized, web-based registryVerified postcodeLondon, United Kingdom· Not yet recruiting
Common questions
What is Fabry disease?
Fabry disease is a rare genetic condition that can affect many parts of the body, causing a range of symptoms.
What is Elfabrio?
Elfabrio is a medicine used to treat Fabry disease. This study is specifically looking at its use around pregnancy and breastfeeding.
Will I get new medicine if I join this study?
No, this is an observational study. You will continue with your usual medical care and any medicines prescribed by your doctor.
How long will I be involved in the study?
You and your baby will be followed from the time you enroll until your baby is 12 months old.
Who can enrol me in the study?
Your doctor can enrol you, or in some places, you might be able to enrol yourself directly.
How to find out more
Chiesi Clinical Trial
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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