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NCT00567073 Clinical Trial

Pompe Pregnancy Sub-Registry

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

Clinical Trial Details — Status: Recruiting

Administrative data
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

Study information
Verified date April 2024
Source Sanofi
Contact Trial Transparency email recommended (Toll free number for US &
Phone 800-633-1610
Email Contact-Us@sanofi.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial 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.

Description:

Study Design Time Perspective: Retrospective and Prospective

Recruitment information / eligibility
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

Study Design

Related Conditions & MeSH terms

Locations
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

Sponsors (1)
Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Countries where clinical trial is conducted

United States,  Belgium,  Croatia,  Czechia,  Italy, 

Outcome
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
See also
  Status Clinical Trial Phase
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Recruiting NCT04174105 - Gene Transfer Study in Patients With Late Onset Pompe Disease Phase 1/Phase 2
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT03285126 - Pompe Gene Therapy- Screening for Eligibility
Recruiting NCT05083806 - MSOT in Pompe Disease N/A
Recruiting NCT05272969 - Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
Active, not recruiting NCT03694561 - Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
Completed NCT00455195 - Late-Onset Treatment Study Extension Protocol Phase 4
Completed NCT00731081 - Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme® N/A
Recruiting NCT05951790 - Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease N/A
Completed NCT01288027 - Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Phase 4
Completed NCT03893240 - Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease N/A
Withdrawn NCT04094948 - Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease Phase 2
Completed NCT00250939 - A Study of rhGAA in Patients With Late-Onset Pompe Disease Phase 2
Recruiting NCT03911505 - ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD Phase 3
Recruiting NCT06150820 - A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease N/A
Completed NCT03729362 - A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Phase 3
Not yet recruiting NCT06178432 - Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD) Early Phase 1
Completed NCT00268944 - Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support Phase 3
Active, not recruiting NCT04138277 - A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD Phase 3