An observational study in Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-onset) and Glycogenesis 2 Acid Maltase Deficiency, sponsored by Genzyme, a Sanofi Company. Recruiting at 35 sites in 9 countries. Open to female participants. Per ClinicalTrials.gov, last updated 2026-04-16.
Sponsored by Genzyme, a Sanofi Company (part of Sanofi) · Observational
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.
Study Design Time Perspective: Retrospective and Prospective
162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.
This study's planned enrollment of 20 is below the median of 50 across 64 observational studies indexed under Glycogen Storage Disease Type II.
Browse Glycogen Storage Disease Type II studies →Genzyme, a Sanofi Company is the lead sponsor of 308 studies on the registry; 5 are open to participants now.
Of its 25 completed or terminated interventional studies of FDA-regulated products, 19 (76%) have results posted.
First submitted before Genzyme became part of Sanofi.
Counted across the registry records on this site, refreshed daily.
Pregnant Females with Pompe disease and/or infants born to females with Pompe disease. Participants may or may not be receiving therapy
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Eligible women must:
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
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
No experimental intervention is given. Pregnant women with confirmed diagnosis of Pompe disease that are participating in the Pompe Registry (NCT00231400) and consented to participate in the Pompe Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglusidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which she may be treated.
Pregnant women with Pompe disease enrolled in the Pompe disease registry (NCT00231400) who are not receiving treatment
The infants of mothers with Pompe disease enrolled in the Pompe disease registry (NCT00231400) where the mothers are receiving treatment of alglucosidase alfa (Myozyme/Lumizyme) or avalglucosidase alfa (Nexviadyme/Nexviazyme)
The infants of mothers with Pompe disease enrolled in the Pompe Disease Registry (NCT00231400) where the mothers are not receiving Treatment
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
Time frame: 10 Months
Follow-up of infants born to women with Pompe disease for 3 years post-partum
Time frame: 3 years
Not enrolling at this site — choose another above.
Started Jun 2007 (actual). Primary completion is estimated for Jan 2034, 319 months after the start.
Of 29 observational studies under Glycogen Storage Disease Type II, half reached primary completion within 108 months of starting (the median), counting those still running; a quarter within 33. This study's planned 319 months is above that.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Glycogen Storage Disease Type II →