Pompe Disease (Late-onset) Clinical Trial
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
NCT number | NCT00567073 |
Other study ID # | AGLU03506 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | June 18, 2007 |
Est. completion date | January 31, 2034 |
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.
Status | Recruiting |
Enrollment | 20 |
Est. completion date | January 31, 2034 |
Est. primary completion date | January 31, 2034 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | N/A and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
Belgium | Investigational Site Number : 056001 | Gent | |
Croatia | Investigational Site Number : 1910001 | Zagreb | |
Croatia | Investigational Site Number : 1910002 | Zagreb | |
Czechia | Investigational Site Number : 2030001 | Praha | |
Italy | Investigational Site Number : 380008 | Brescia | |
Italy | Investigational Site Number : 380006 | Cagliari | |
Italy | Investigational Site Number : 380005 | Firenze | |
Italy | Investigational Site Number : 380004 | Genova | |
Italy | Investigational Site Number : 380013 | Messina | |
Italy | Investigational Site Number : 380007 | Milano | |
Italy | Investigational Site Number : 380009 | Monza | |
Italy | Investigational Site Number : 380002 | Padova | |
Italy | Investigational Site Number : 380011 | Padova | |
Italy | Investigational Site Number : 380003 | Pavia | |
Italy | Investigational Site Number : 380012 | Roma | |
Italy | Investigational Site Number : 380015 | Roma | |
United States | Emory University School Of Medicine Site Number : 840060 | Atlanta | Georgia |
United States | LSD Data Registry Site LLC Site Number : 840094 | Dublin | Ohio |
United States | Duke University Medical Center Genetics Dept Site Number : 840037 | Durham | North Carolina |
United States | O&O Alpan, LLC Site Number : 840025 | Fairfax | Virginia |
United States | Spectrum for Health Site Number : 840019 | Grand Rapids | Michigan |
United States | Indianapolis University School of Medicine Site Number : 840027 | Indianapolis | Indiana |
United States | Mt. Sinai School of Medicine Site Number : 840005 | New York | New York |
United States | New York University School Of Medicine Site Number : 840040 | New York | New York |
United States | Barrow Neurol Group Site Number : 840087 | Phoenix | Arizona |
United States | Oregon Health and Science University Site Number : 840095 | Portland | Oregon |
Lead Sponsor | Collaborator |
---|---|
Genzyme, a Sanofi Company |
United States, Belgium, Croatia, Czechia, Italy,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | ||
Primary | Follow-up of infants born to women with Pompe disease for 3 years post-partum | 3 years |
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