Pompe Pregnancy Sub-Registry
- Conditions
- Glycogenesis 2 Acid Maltase DeficiencyGlycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)
- Registration Number
- NCT00567073
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief 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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Female
- Target Recruitment
- 20
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.
There are no exclusion criteria for this Sub-Registry
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Follow-up of infants born to women with Pompe disease for 3 years post-partum 3 years 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 10 Months
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (26)
Barrow Neurol Group- Site Number : 840087
🇺🇸Phoenix, Arizona, United States
Investigational Site Number : 380013
🇮🇹Messina, Italy
Investigational Site Number : 380007
🇮🇹Milan, Italy
Investigational Site Number : 380009
🇮🇹Monza, Italy
Investigational Site Number : 380002
🇮🇹Padova, Italy
Investigational Site Number : 380011
🇮🇹Padua, Italy
Investigational Site Number : 380003
🇮🇹Pavia, Italy
Investigational Site Number : 380015
🇮🇹Roma, Italy
Investigational Site Number : 380012
🇮🇹Roma, Italy
Emory University School Of Medicine- Site Number : 840060
🇺🇸Atlanta, Georgia, United States
Indianapolis University School of Medicine- Site Number : 840027
🇺🇸Indianapolis, Indiana, United States
Spectrum for Health- Site Number : 840019
🇺🇸Grand Rapids, Michigan, United States
New York University School Of Medicine- Site Number : 840040
🇺🇸New York, New York, United States
Mt. Sinai School of Medicine- Site Number : 840005
🇺🇸New York, New York, United States
Duke University Medical Center Genetics Dept- Site Number : 840037
🇺🇸Durham, North Carolina, United States
LSD Data Registry Site LLC- Site Number : 840094
🇺🇸Dublin, Ohio, United States
Oregon Health and Science University- Site Number : 840095
🇺🇸Portland, Oregon, United States
O&O Alpan, LLC- Site Number : 840025
🇺🇸Fairfax, Virginia, United States
Investigational Site Number : 056001
🇧🇪Gent, Belgium
Investigational Site Number : 1910001
🇭🇷Zagreb, Croatia
Investigational Site Number : 1910002
🇭🇷Zagreb, Croatia
Investigational Site Number : 2030001
🇨🇿Praha, Czechia
Investigational Site Number : 380008
🇮🇹Brescia, Italy
Investigational Site Number : 380006
🇮🇹Cagliari, Italy
Investigational Site Number : 380005
🇮🇹Firenze, Italy
Investigational Site Number : 380004
🇮🇹Genova, Italy