ClinicalTrials.gov record NCT00567073
Pompe Pregnancy Sub-Registry
A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This voluntary sub-registry collects medical information on pregnancies and infants of women enrolled in the Pompe Registry. It includes both past (retrospective) and future (prospective) pregnancies and records outcomes, complications, and infant growth up to three years. No experimental treatment is given; participants continue their usual clinical care.
What it is studying. The registry aims to observe and record pregnancy outcomes, pregnancy-related complications, and infant growth in women with Pompe disease, and to observe these outcomes whether or not the mother receives alglucosidase alfa or avalglucosidase alfa treatment.
What the researchers hope to learn. To track pregnancy outcomes and complications in women with Pompe disease; to track infant growth up to three years; to observe outcomes regardless of whether mothers receive alglucosidase alfa or avalglucosidase alfa.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Pompe disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with alglucosidase alfa or avalglucosidase alfa.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Eligible women must:
- be enrolled in the Pompe registry (NCT00231400)
- be pregnant, or have been pregnant with appropriate medical documentation available.
- provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.
- Note: It is recommended that pregnancy data be collected on eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, participation in this Sub-Registry is encouraged for each individual pregnancy.
Exclusion criteria
- There are no exclusion criteria for this Sub-Registry
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
Study Design Time Perspective: Retrospective and Prospective
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants allow the study team to collect pregnancy and delivery medical records and information on pregnancy complications and infant growth, using past records or ongoing follow-up (retrospective and prospective data). No experimental treatment is given; participants continue standard care and any Pompe-specific therapy as decided by their physician. Infants born to enrolled mothers may be followed for up to 3 years after birth; overall data collection is planned across about 4 years. You must sign informed consent before any Sub-Registry-related data are collected.
How the study is structured. A voluntary, longitudinal registry that collects existing medical records and ongoing clinical information from women in the Pompe Registry. It includes both retrospective and prospective data collection, follows pregnancies through delivery, and follows infants for up to three years postpartum. No study-assigned treatments are given; care is determined by each patient's clinician.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Australia, Belgium, Brazil, Croatia, Czechia, Germany, Italy, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Pompe disease
Important dates
- Study start
- Jun 18, 2007
- Primary completion
- Jan 31, 2034 (estimated)
- Study completion
- Jan 31, 2034 (estimated)
- Target enrollment
- 20 participants (estimated)
- Last updated on registry
- Apr 16, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Genzyme, a Sanofi Company
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Trial Transparency email recommended (Toll free number for US & Canada) 📞 800-633-1610 ext. option 6 ✉ Contact-us@sanofi.com
- Pompe Registry HelpLine 📞 617-591-5500
Participating sites (35)
- Barrow Neurol Group- Site Number : 840087Recruiting
Phoenix, Arizona, 85013 · United States
- University of Florida Pediatrics Genetics- Site Number : 157138Recruiting
Jacksonville, Florida, 32207 · United States
- Emory University School Of Medicine- Site Number : 840060Recruiting
Atlanta, Georgia, 30322 · United States
- Indianapolis University School of Medicine- Site Number : 840027Recruiting
Indianapolis, Indiana, 46202 · United States
- University of Iowa- Site Number : 150294Recruiting
Iowa City, Iowa, 52242-1009 · United States
- Spectrum for Health- Site Number : 840019Recruiting
Grand Rapids, Michigan, 49503 · United States
- Washington University- Site Number : 150612Recruiting
St Louis, Missouri, 63110 · United States
- New York University School Of Medicine- Site Number : 840040Recruiting
New York, New York, 10016 · United States
- Mt. Sinai School of Medicine- Site Number : 840005Recruiting
New York, New York, 10029 · United States
- Columbia University Irving Medical Center- Site Number : 157199Recruiting
New York, New York, 10032 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- registry
- An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
- standard of care
- Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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