Trials / Recruiting
RecruitingNCT00567073
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
- Status
- Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 20 (estimated)
- Sponsor
- Genzyme, a Sanofi Company · Industry
- Sex
- Female
- Age
- —
- Healthy volunteers
- Not accepted
Summary
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.
Detailed description
Study Design Time Perspective: Retrospective and Prospective
Conditions
- Glycogen Storage Disease Type II (GSD-II)
- Pompe Disease (Late-onset)
- Glycogenesis 2 Acid Maltase Deficiency
Timeline
- Start date
- 2007-06-18
- Primary completion
- 2034-01-31
- Completion
- 2034-01-31
- First posted
- 2007-12-04
- Last updated
- 2026-04-16
Locations
35 sites across 9 countries: United States, Australia, Belgium, Brazil, Croatia, Czechia, Germany, Italy, United Kingdom
Source: ClinicalTrials.gov record NCT00567073. Inclusion in this directory is not an endorsement.