Clinical trial NCT00567073: Pompe Pregnancy Sub-Registry
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Glycogen Storage Disease Type II (GSD-II)
Pompe Disease (Late-onset)
Glycogenesis 2 Acid Maltase Deficiency
Keywords: Glycogen Storage Disease Type II (GSD-II)
GSD-II
Pompe Disease
Pompe Disease (Late-Onset)
Acid Maltase Deficiency Disease
Glycogenosis II
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2007-12-04
Enrollment: 20
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pregnant Females with Pompe disease and/or infants born to females with Pompe disease. Participants may or may not be receiving therapy

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

Eligibility criteria: 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 outcomes:
measure: 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
timeFrame: 10 Months
measure: Follow-up of infants born to women with Pompe disease for 3 years post-partum
timeFrame: 3 years
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurol Group- Site Number : 840087
state: Arizona
city: Atlanta
country: United States
facility: Emory University School Of Medicine- Site Number : 840060
state: Georgia
city: Indianapolis
country: United States
facility: Indianapolis University School of Medicine- Site Number : 840027
state: Indiana
city: Grand Rapids
country: United States
facility: Spectrum for Health- Site Number : 840019
state: Michigan
city: New York
country: United States
facility: New York University School Of Medicine- Site Number : 840040
state: New York
city: New York
country: United States
facility: Mt. Sinai School of Medicine- Site Number : 840005
state: New York
city: Durham
country: United States
facility: Duke University Medical Center Genetics Dept- Site Number : 840037
state: North Carolina
city: Dublin
country: United States
facility: LSD Data Registry Site LLC- Site Number : 840094
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University- Site Number : 840095
state: Oregon
city: Fairfax
country: United States
facility: O&O Alpan, LLC- Site Number : 840025
state: Virginia
city: Gent
country: Belgium
facility: Investigational Site Number : 056001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910001
city: Zagreb
country: Croatia
facility: Investigational Site Number : 1910002
city: Praha
country: Czechia
facility: Investigational Site Number : 2030001
city: Brescia
country: Italy
facility: Investigational Site Number : 380008
city: Cagliari
country: Italy
facility: Investigational Site Number : 380006
city: Firenze
country: Italy
facility: Investigational Site Number : 380005
city: Genova
country: Italy
facility: Investigational Site Number : 380004
city: Messina
country: Italy
facility: Investigational Site Number : 380013
city: Milan
country: Italy
facility: Investigational Site Number : 380007
city: Monza
country: Italy
facility: Investigational Site Number : 380009
city: Padova
country: Italy
facility: Investigational Site Number : 380002
city: Padua
country: Italy
facility: Investigational Site Number : 380011
city: Pavia
country: Italy
facility: Investigational Site Number : 380003
city: Roma
country: Italy
facility: Investigational Site Number : 380015
city: Roma
country: Italy
facility: Investigational Site Number : 380012
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Study Director
role: STUDY_DIRECTOR
References: