RecruitingOBSERVATIONAL

Pompe Pregnancy Sub-Registry

This research project, called the Pompe Pregnancy Sub-Registry, is designed to keep track of how pregnancies go for women who have Pompe disease. It's an international study that follows women over time, collecting information about their health during pregnancy and the well-being of their babies. It doesn't involve giving new medicines or treatments; instead, it observes what happens when women receive standard care from their doctors, including any existing Pompe disease treatments. The main goal is to understand better how pregnancy impacts women with Pompe disease and to learn about any complications or how babies grow, whether the mother is on specific Pompe medication or not.

At a glance

Status

Recruiting

Sponsor

Genzyme, a Sanofi Company

Enrolment target

Start

18 Jun 2007

Estimated completion

31 Jan 2034

Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency

What is this study about?

Imagine a special notebook where doctors and researchers write down all the important details about pregnant women with Pompe disease. That's essentially what the Pompe Pregnancy Sub-Registry is. It's like a long-term look at how pregnancy goes for these women, whether they are taking medicine for their Pompe disease or not. The doctors and nurses involved will simply record information that’s already collected as part of the woman’s normal care, with her permission, rather than asking for any extra tests or treatments.

The main idea behind this study is to gather as much information as possible about pregnancy in women with Pompe disease. This includes looking at things like how the pregnancy develops, any health issues that might come up for the mother, and how her baby grows. By collecting all this information from many different women across different countries, researchers hope to get a clearer picture of how Pompe disease and its treatments might affect pregnancy and new babies.

This kind of study is really important because it helps specialist doctors learn more about the best ways to support women with Pompe disease who become pregnant. It aims to improve understanding for future mothers and their healthcare teams, ultimately helping to ensure the safest and healthiest outcomes for both mum and baby.

Key takeaways

It's an observational study, no new treatments.
Aims to understand pregnancy in women with Pompe disease.
Collects existing medical record data about mum and baby.
Helps improve care for future pregnant mothers with Pompe disease.
For women already in the Pompe Registry, who are pregnant or have been.

Who may be eligible?

You might be able to take part in this study if you are a woman who has Pompe disease and you are currently pregnant, or you have been pregnant in the past and still have your medical records available for that pregnancy.

To join, you also need to be already signed up to a larger existing study called the Pompe Registry. Once you meet these criteria, you would need to give your official permission by signing a consent form to allow your pregnancy information to be included in this specific study.

There aren't any other strict rules that would stop you from joining, which means if you meet the points above, you're likely eligible. If you have had more than one pregnancy, they would be interested in collecting information about each one separately.

Quick self-check

Are you a woman with Pompe disease?
Are you currently pregnant or have you been pregnant in the past?
Do you have medical records available for that pregnancy?
Are you already signed up for the main Pompe Registry study?
Are you willing to sign a consent form?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you won't be given any new or experimental medicines. You will continue to have your usual check-ups, tests, and treatments for Pompe disease and your pregnancy, as decided by your own doctor. The study team will simply collect information from your existing medical records regarding your pregnancy, any treatments you might be on for Pompe disease (like alglucosidase alfa or avalglucosidase alfa), and the health of your baby.

This is an ongoing study, and your involvement would last for the duration of your pregnancy and potentially for some time afterwards to track your baby's health. The study team may ask to collect information for each pregnancy you have, if you have more than one. You would need to sign a consent form before any information is collected.

Potential risks and benefits

Because this study only involves collecting information from your existing medical records and doesn't ask you to undergo any new tests or treatments, there are no direct medical risks to you or your baby. The information gathered may help improve care for other pregnant women with Pompe disease in the future. The main benefit is contributing to a better understanding of Pompe disease during pregnancy. You are free to withdraw your consent and participation at any time, without needing to give a reason, and this would not affect your medical care.

Locations (35)

Barrow Neurol Group- Site Number : 840087
Phoenix, United States· Recruiting
University of Florida Pediatrics Genetics- Site Number : 157138
Jacksonville, United States· Recruiting
Emory University School Of Medicine- Site Number : 840060
Atlanta, United States· Recruiting
Indianapolis University School of Medicine- Site Number : 840027
Indianapolis, United States· Recruiting
University of Iowa- Site Number : 150294
Iowa City, United States· Recruiting
Spectrum for Health- Site Number : 840019
Grand Rapids, United States· Recruiting
Washington University- Site Number : 150612
St Louis, United States· Recruiting
New York University School Of Medicine- Site Number : 840040
New York, United States· Recruiting
Mt. Sinai School of Medicine- Site Number : 840005
New York, United States· Recruiting
Columbia University Irving Medical Center- Site Number : 157199
New York, United States· Recruiting
Duke University Medical Center Genetics Dept- Site Number : 840037
Durham, United States· Recruiting
LSD Data Registry Site LLC- Site Number : 840094
Dublin, United States· Recruiting

+23 more sites — see the official record for the full list.

Common questions

What is Pompe disease?

Pompe disease is a rare genetic condition where the body can't properly break down a complex sugar called glycogen, which then builds up and can cause problems in muscles and other organs.

Will I have to take new medicine or get extra tests?

No, you won't be given any new medicine or asked to have extra tests for this study. It only collects information from your existing medical records.

What kind of information will be collected?

The study will collect details about your pregnancy, any treatments you receive for Pompe disease, and how your baby is growing and developing.

Who can join this study?

Women with Pompe disease who are pregnant (or have been pregnant) and are already part of the main Pompe Registry can join, after giving their permission.

Will my information be kept private?

Yes, all your personal and medical information will be kept strictly confidential and anonymous when used for research purposes, meaning your name won't be connected to the data.

How to find out more

Trial Transparency email recommended (Toll free number for US & Canada)

Contact-us@sanofi.com 800-633-1610 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.