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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00196742




Registration number
NCT00196742
Ethics application status
Date submitted
13/09/2005
Date registered
20/09/2005
Date last updated
20/11/2024

Titles & IDs
Public title
Fabry Disease Registry & Pregnancy Sub-registry
Scientific title
Fabry Disease Registry Protocol
Secondary ID [1] 0 0
AGAL19211
Secondary ID [2] 0 0
DIREGC07006
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Patients with Fabry disease - No experimental intervention is given. A patient with Fabry Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.

Pregnant women with confirmed diagnosis of Fabry - No experimental intervention is given. Pregnant women with confirmed diagnosis of Fabry that are participating in the Fabry Registry and consented to participate in the Fabry Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with agalsidase beta) and irrespective of the commercial product with which she may be treated.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme®
Timepoint [1] 0 0
33 years
Primary outcome [2] 0 0
Fabry Pregnancy Sub-registry: pregnancy outcomes, including complications and infant growth
Timepoint [2] 0 0
33 years
Secondary outcome [1] 0 0
Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments
Timepoint [1] 0 0
33 years

Eligibility
Key inclusion criteria
Inclusion Criteria

* Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte aGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for aGAL.
* Fabry Pregnancy Sub-registry:

* Eligible women must:

* be enrolled in the Fabry Registry.
* 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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.

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
31/07/2001
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/01/2034
Actual
Sample size
Target
9000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Investigational Site Number : 036013 - Westmead
Recruitment hospital [2] 0 0
Investigational Site Number : 151069 - Westmead
Recruitment hospital [3] 0 0
Investigational Site Number : 153063 - Brisbane
Recruitment hospital [4] 0 0
Investigational Site Number : 151002 - Adelaide
Recruitment hospital [5] 0 0
Investigational Site Number : 153040 - Parkville
Recruitment hospital [6] 0 0
Investigational Site Number : 153090 - Perth
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4029 - Brisbane
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment postcode(s) [4] 0 0
3050 - Parkville
Recruitment postcode(s) [5] 0 0
6000 - Perth
Recruitment outside Australia
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United States of America
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Alabama
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Belfast
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Birmingham
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Salford
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Vietnam
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Hanoi

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

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
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT00196742