Watching how pregnancy goes for women living with Pompe disease
Follows pregnancies and babies' growth in mothers with Pompe disease.
Key facts
- Who runs it
- Genzyme, a Sanofi Company
- Where
- São Paulo, BrazilSee the siteAlso in 8 other countries, 34 sites
- Trial code
- Registry title
- Pompe Pregnancy Sub-Registry
- Status
- Recruiting
- People taking part
- 20 people, planned
- Started
- 18 Jun 2007
- Expected to end
- 31 Jan 2034
- Type of study
- Observational, 4 groups
- Record updated
- 16 April 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for women with Pompe disease who are already in the Pompe Registry and are pregnant now or have been pregnant before.
- Sex
- Women only.
- Disease stage
- Women with Pompe disease. The record gives no stage or severity.
- Earlier treatment
- The record sets no treatment requirement. Women can join whether or not they receive Pompe therapy.
The registry criteria, word for word
Show all 5 criteriaShow less
Who can take part
- 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
Who cannot take part
- There are no exclusion criteria for this Sub-Registry
Where in Brazil
Also in 8 other countries, 34 sites
- Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 150978São Paulo, BrazilContact: Pompe Registry HelpLineTaking people
About the trial
What the trial wants to find out
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.
Read full original descriptionShow less
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.
The 4 groups in the trial
- Pregnant women with confirmed diagnosis of Pompe Disease
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 receiving no treatment for Pompe disease
Pregnant women with Pompe disease enrolled in the Pompe disease registry (NCT00231400) who are not receiving treatment
- Infants born to mothers receiving treatment for Pompe disease
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)
- Infants born to mothers receiving no treatment for Pompe disease
The infants of mothers with Pompe disease enrolled in the Pompe Disease Registry (NCT00231400) where the mothers are not receiving Treatment
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- A Sub-registry to Observe the Effect of Alglucosidase Alfa or Avalglucosidase Alfa Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
- Plain-language title
- Watching how pregnancy goes for women living with Pompe diseaseGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Record updated
- 16 April 2026