Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00567073




Registration number
NCT00567073
Ethics application status
Date submitted
1/12/2007
Date registered
4/12/2007
Date last updated
21/09/2023

Titles & IDs
Public title
Pompe Pregnancy Sub-Registry
Scientific title
A Sub-Registry to Observe the Effect of Myozyme (Alglucosidase Alfa) Treatment on Pregnancy and Infant Growth in Women With Pompe Disease
Secondary ID [1] 0 0
AGLU03506
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Glycogen Storage Disease Type II (GSD-II) 0 0
Pompe Disease (Late-onset) 0 0
Glycogenesis 2 Acid Maltase Deficiency 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Pregnant women with confirmed diagnosis of pompe Disease - No experimental intervention is given. Pregnant women with confirmed diagnosis of Pompe disease that are participating in the Pompe Registry (NCT00231400) and consented to participate in the Pompe Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglusidase alfa) and irrespective of the commercial product with which she may be treated.

Pregnant women receiving no treatment for pompe disease - Pregnant women with pompe disease enrolled in the pompe disease registry (NCT00231400) who are not receiving treatment

Infants born to mothers receiving treatment for pompe disease - The infants of mothers with pompe disease enrolled in the pompe disease registry (NCT00231400) where the mothers are receiving treatment of alglucosidase alpha (Myozyme)

Infants born to mothers receiving no treatment for pompe disease - The infants of mothers with pompe disease enrolled in the Pompe Disease Registry (NCT00231400) where the mothers are not receiving Treatment
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
10 Months
Primary outcome [2] 0 0
Follow-up of infants born to women with Pompe disease for 3 years post-partum
Timepoint [2] 0 0
3 years

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Sex
Females
Can healthy volunteers participate?
No
Key exclusion criteria
There are no exclusion criteria for this Sub-Registry

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/11/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/01/2034
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Oregon
Country [9] 0 0
United States of America
State/province [9] 0 0
Virginia
Country [10] 0 0
Belgium
State/province [10] 0 0
Gent
Country [11] 0 0
Croatia
State/province [11] 0 0
Zagreb
Country [12] 0 0
Italy
State/province [12] 0 0
Brescia
Country [13] 0 0
Italy
State/province [13] 0 0
Cagliari
Country [14] 0 0
Italy
State/province [14] 0 0
Firenze
Country [15] 0 0
Italy
State/province [15] 0 0
Genova
Country [16] 0 0
Italy
State/province [16] 0 0
Messina
Country [17] 0 0
Italy
State/province [17] 0 0
Milano
Country [18] 0 0
Italy
State/province [18] 0 0
Monza
Country [19] 0 0
Italy
State/province [19] 0 0
Padova
Country [20] 0 0
Italy
State/province [20] 0 0
Pavia
Country [21] 0 0
Italy
State/province [21] 0 0
Roma

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Genzyme, a Sanofi Company
Address
Country

Ethics approval
Ethics application status

Summary
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) 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00567073
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Genzyme, a Sanofi Company
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Transparency email recommended (Toll free number for US & Canada)
Address 0 0
Country 0 0
Phone 0 0
800-633-1610
Fax 0 0
Email 0 0
Contact-Us@sanofi.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00567073