Pompe Pregnancy Sub-Registry

NCT00567073 · tracked via the Priya Life Science USA tracker

Sponsor

Genzyme, a Sanofi Company

Phase

Observational

Started

2007-06-18

Last updated

2026-04-16

Condition(s) studied

Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency

Study 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.

Eligibility

Sex

FEMALE

Min age

—

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

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
Follow-up of infants born to women with Pompe disease for 3 years post-partum — 3 years

Trial sites (35)

Facility	City	Region	Status
Barrow Neurol Group- Site Number : 840087	Phoenix	Arizona	Recruiting
University of Florida Pediatrics Genetics- Site Number : 157138	Jacksonville	Florida	Recruiting
Emory University School Of Medicine- Site Number : 840060	Atlanta	Georgia	Recruiting
Indianapolis University School of Medicine- Site Number : 840027	Indianapolis	Indiana	Recruiting
University of Iowa- Site Number : 150294	Iowa City	Iowa	Recruiting
Spectrum for Health- Site Number : 840019	Grand Rapids	Michigan	Recruiting
Washington University- Site Number : 150612	St Louis	Missouri	Recruiting
New York University School Of Medicine- Site Number : 840040	New York	New York	Recruiting
Mt. Sinai School of Medicine- Site Number : 840005	New York	New York	Recruiting
Columbia University Irving Medical Center- Site Number : 157199	New York	New York	Recruiting
Duke University Medical Center Genetics Dept- Site Number : 840037	Durham	North Carolina	Recruiting
LSD Data Registry Site LLC- Site Number : 840094	Dublin	Ohio	Recruiting
Oregon Health and Science University- Site Number : 840095	Portland	Oregon	Completed
Greenwood Genetic Center - Greenville- Site Number : 151184	Greenville	South Carolina	Recruiting
O & O Alpan- Site Number : 840025	Fairfax	Virginia	Recruiting
Investigational Site Number : 151069	Westmead	New South Wales	Recruiting
Investigational Site Number : 056001	Ghent	Belgium	Recruiting
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 150978	São Paulo	Brazil	Recruiting
Investigational Site Number : 1910001	Zagreb	Croatia	Recruiting
Investigational Site Number : 1910002	Zagreb	Croatia	Recruiting
Investigational Site Number : 2030001	Prague	Czechia	Recruiting
Investigational Site Number : 152200	Bonn	Germany	Recruiting
Investigational Site Number : 380005	Florence	Firenze	Recruiting
Investigational Site Number : 380007	Milan	Milano	Recruiting
Investigational Site Number : 380011	Padua	Padova	Recruiting
Investigational Site Number : 380015	Rome	Roma	Recruiting
Investigational Site Number : 380008	Brescia	Italy	Recruiting
Investigational Site Number : 380006	Cagliari	Italy	Recruiting
Investigational Site Number : 380004	Genova	Italy	Recruiting
Investigational Site Number : 380013	Messina	Italy	Recruiting
Investigational Site Number : 380009	Monza	Italy	Recruiting
Investigational Site Number : 380002	Padova	Italy	Recruiting
Investigational Site Number : 380003	Pavia	Italy	Recruiting
Investigational Site Number : 380012	Roma	Italy	Recruiting
Investigational Site Number : 87147	Cardiff	Cardiff [Caerdydd Gb-crd]	Recruiting

More Genzyme, a Sanofi Company trials in the USA

Pompe Disease Registry Protocol Recruiting

Fabry Disease Registry & Pregnancy Sub-registry Recruiting

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension Active, not recruiting

A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa Active, not recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry Recruiting

Other trials for the same condition

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) Recruiting

Gene Transfer Study in Patients With Late Onset Pompe Disease Active, not recruiting

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease Active, not recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Active, not recruiting

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT00567073 on ClinicalTrials.gov ↗ ← All trials in the USA