Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
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
17/04/2024
Titles & IDs
Public title
Fabry Disease Registry & Pregnancy Sub-registry
Query!
Scientific title
Fabry Disease Registry Protocol
Query!
Secondary ID [1]
0
0
AGAL19211
Query!
Secondary ID [2]
0
0
DIREGC07006
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Metabolic disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Intervention/exposure
Study type
Observational [Patient Registry]
Query!
Patient registry
Query!
Target follow-up duration
Query!
Target follow-up type
Query!
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.
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme®
Query!
Assessment method [1]
0
0
The primary purpose of this Registry is to describe the development and progression of Fabry disease in a representative global population. As Fabry is not a well-described disease this longitudinal program has a wide variety of "primary" outcomes including antibody testing as well as complication outcomes (eg, QoL, cognitive testing) which are measured over time. Additionally, as it is subject to what is collected by clinical sites at the time of visit it is unknown the amount of data that will be available for analyses.
Query!
Timepoint [1]
0
0
33 years
Query!
Primary outcome [2]
0
0
Fabry Pregnancy Sub-registry: pregnancy outcomes, including complications and infant growth
Query!
Assessment method [2]
0
0
The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Fabry disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with agalsidase beta.
This Sub-registry augments the routine monitoring and data collection recommended by the Fabry Registry. The Sub-registry aims to collect patient assessments from the ante- and perinatal periods and postpartum follow-up. Neonatal assessments and periodic pediatric assessments will also be collected.
Query!
Timepoint [2]
0
0
33 years
Query!
Secondary outcome [1]
0
0
Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments
Query!
Assessment method [1]
0
0
A secondary endpoint is to monitor those factors associated with the efficacy of Fabry disease treatments.
Query!
Timepoint [1]
0
0
33 years
Query!
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.
Query!
Minimum age
No limit
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
Exclusion Criteria Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.
Query!
Study design
Purpose
Query!
Duration
Query!
Selection
Query!
Timing
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
31/07/2001
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
31/01/2034
Query!
Actual
Query!
Sample size
Target
9000
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Query!
Recruitment hospital [1]
0
0
Investigational Site Number : 036013 - Westmead
Query!
Recruitment hospital [2]
0
0
Investigational Site Number : 151069 - Westmead
Query!
Recruitment hospital [3]
0
0
Investigational Site Number : 153063 - Herston
Query!
Recruitment hospital [4]
0
0
Investigational Site Number : 151002 - Adelaide
Query!
Recruitment hospital [5]
0
0
Investigational Site Number : 153040 - Parkville
Query!
Recruitment hospital [6]
0
0
Investigational Site Number : 153090 - Perth
Query!
Recruitment postcode(s) [1]
0
0
2145 - Westmead
Query!
Recruitment postcode(s) [2]
0
0
4006 - Herston
Query!
Recruitment postcode(s) [3]
0
0
5000 - Adelaide
Query!
Recruitment postcode(s) [4]
0
0
3050 - Parkville
Query!
Recruitment postcode(s) [5]
0
0
6000 - Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Alabama
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Arizona
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Arkansas
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
California
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Colorado
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Connecticut
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
District of Columbia
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Florida
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Georgia
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Illinois
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Indiana
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Iowa
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Kansas
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Kentucky
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Louisiana
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Maine
Query!
Country [17]
0
0
United States of America
Query!
State/province [17]
0
0
Maryland
Query!
Country [18]
0
0
United States of America
Query!
State/province [18]
0
0
Massachusetts
Query!
Country [19]
0
0
United States of America
Query!
State/province [19]
0
0
Michigan
Query!
Country [20]
0
0
United States of America
Query!
State/province [20]
0
0
Minnesota
Query!
Country [21]
0
0
United States of America
Query!
State/province [21]
0
0
Missouri
Query!
Country [22]
0
0
United States of America
Query!
State/province [22]
0
0
Montana
Query!
Country [23]
0
0
United States of America
Query!
State/province [23]
0
0
Nebraska
Query!
Country [24]
0
0
United States of America
Query!
State/province [24]
0
0
Nevada
Query!
Country [25]
0
0
United States of America
Query!
State/province [25]
0
0
New Jersey
Query!
Country [26]
0
0
United States of America
Query!
State/province [26]
0
0
New York
Query!
Country [27]
0
0
United States of America
Query!
State/province [27]
0
0
North Carolina
Query!
Country [28]
0
0
United States of America
Query!
State/province [28]
0
0
Ohio
Query!
Country [29]
0
0
United States of America
Query!
State/province [29]
0
0
Oregon
Query!
Country [30]
0
0
United States of America
Query!
State/province [30]
0
0
Pennsylvania
Query!
Country [31]
0
0
United States of America
Query!
State/province [31]
0
0
Rhode Island
Query!
Country [32]
0
0
United States of America
Query!
State/province [32]
0
0
South Carolina
Query!
Country [33]
0
0
United States of America
Query!
State/province [33]
0
0
Tennessee
Query!
Country [34]
0
0
United States of America
Query!
State/province [34]
0
0
Texas
Query!
Country [35]
0
0
United States of America
Query!
State/province [35]
0
0
Utah
Query!
Country [36]
0
0
United States of America
Query!
State/province [36]
0
0
Virginia
Query!
Country [37]
0
0
United States of America
Query!
State/province [37]
0
0
Washington
Query!
Country [38]
0
0
United States of America
Query!
State/province [38]
0
0
Wisconsin
Query!
Country [39]
0
0
Argentina
Query!
State/province [39]
0
0
Buenos Aires
Query!
Country [40]
0
0
Argentina
Query!
State/province [40]
0
0
Catamarca
Query!
Country [41]
0
0
Argentina
Query!
State/province [41]
0
0
La Rioja
Query!
Country [42]
0
0
Argentina
Query!
State/province [42]
0
0
Santa Fe
Query!
Country [43]
0
0
Belgium
Query!
State/province [43]
0
0
Antwerpen
Query!
Country [44]
0
0
Belgium
Query!
State/province [44]
0
0
Brussels
Query!
Country [45]
0
0
Belgium
Query!
State/province [45]
0
0
Bruxelles
Query!
Country [46]
0
0
Belgium
Query!
State/province [46]
0
0
Jette
Query!
Country [47]
0
0
Belgium
Query!
State/province [47]
0
0
Leuven
Query!
Country [48]
0
0
Belgium
Query!
State/province [48]
0
0
Liège
Query!
Country [49]
0
0
Belgium
Query!
State/province [49]
0
0
Seraing
Query!
Country [50]
0
0
Brazil
Query!
State/province [50]
0
0
Bahia
Query!
Country [51]
0
0
Brazil
Query!
State/province [51]
0
0
Distrito Federal
Query!
Country [52]
0
0
Brazil
Query!
State/province [52]
0
0
Mato Grosso
Query!
Country [53]
0
0
Brazil
Query!
State/province [53]
0
0
Minas Gerais
Query!
Country [54]
0
0
Brazil
Query!
State/province [54]
0
0
Paraná
Query!
Country [55]
0
0
Brazil
Query!
State/province [55]
0
0
Pernambuco
Query!
Country [56]
0
0
Brazil
Query!
State/province [56]
0
0
Rio Grande Do Sul
Query!
Country [57]
0
0
Brazil
Query!
State/province [57]
0
0
Santa Catarina
Query!
Country [58]
0
0
Brazil
Query!
State/province [58]
0
0
São Paulo
Query!
Country [59]
0
0
Bulgaria
Query!
State/province [59]
0
0
Sofia
Query!
Country [60]
0
0
Canada
Query!
State/province [60]
0
0
Alberta
Query!
Country [61]
0
0
Canada
Query!
State/province [61]
0
0
British Columbia
Query!
Country [62]
0
0
Canada
Query!
State/province [62]
0
0
Manitoba
Query!
Country [63]
0
0
Canada
Query!
State/province [63]
0
0
New Brunswick
Query!
Country [64]
0
0
Canada
Query!
State/province [64]
0
0
Newfoundland and Labrador
Query!
Country [65]
0
0
Canada
Query!
State/province [65]
0
0
Nova Scotia
Query!
Country [66]
0
0
Canada
Query!
State/province [66]
0
0
Ontario
Query!
Country [67]
0
0
Canada
Query!
State/province [67]
0
0
Quebec
Query!
Country [68]
0
0
Chile
Query!
State/province [68]
0
0
Antofagasta
Query!
Country [69]
0
0
Chile
Query!
State/province [69]
0
0
Coquimbo
Query!
Country [70]
0
0
Chile
Query!
State/province [70]
0
0
Iquique
Query!
Country [71]
0
0
Chile
Query!
State/province [71]
0
0
Vallenar
Query!
Country [72]
0
0
China
Query!
State/province [72]
0
0
Hangzhou
Query!
Country [73]
0
0
China
Query!
State/province [73]
0
0
Jinan
Query!
Country [74]
0
0
China
Query!
State/province [74]
0
0
Nanjing
Query!
Country [75]
0
0
Colombia
Query!
State/province [75]
0
0
Bogotá
Query!
Country [76]
0
0
Croatia
Query!
State/province [76]
0
0
Rijena
Query!
Country [77]
0
0
Croatia
Query!
State/province [77]
0
0
Zagreb
Query!
Country [78]
0
0
Czechia
Query!
State/province [78]
0
0
Praha 2
Query!
Country [79]
0
0
Denmark
Query!
State/province [79]
0
0
Copenhagen
Query!
Country [80]
0
0
Denmark
Query!
State/province [80]
0
0
København Ø
Query!
Country [81]
0
0
Estonia
Query!
State/province [81]
0
0
Tartu
Query!
Country [82]
0
0
Finland
Query!
State/province [82]
0
0
Turku
Query!
Country [83]
0
0
France
Query!
State/province [83]
0
0
Bordeaux
Query!
Country [84]
0
0
France
Query!
State/province [84]
0
0
BRON Cedex
Query!
Country [85]
0
0
France
Query!
State/province [85]
0
0
Garches
Query!
Country [86]
0
0
France
Query!
State/province [86]
0
0
Lille
Query!
Country [87]
0
0
France
Query!
State/province [87]
0
0
LIMOGES Cedex
Query!
Country [88]
0
0
France
Query!
State/province [88]
0
0
Marseille
Query!
Country [89]
0
0
France
Query!
State/province [89]
0
0
Saint Priest en Jarez
Query!
Country [90]
0
0
France
Query!
State/province [90]
0
0
Strasbourg
Query!
Country [91]
0
0
Germany
Query!
State/province [91]
0
0
Berlin
Query!
Country [92]
0
0
Germany
Query!
State/province [92]
0
0
Gießen
Query!
Country [93]
0
0
Germany
Query!
State/province [93]
0
0
Münster
Query!
Country [94]
0
0
Germany
Query!
State/province [94]
0
0
Würzburg
Query!
Country [95]
0
0
Hong Kong
Query!
State/province [95]
0
0
Kowloon
Query!
Country [96]
0
0
Hungary
Query!
State/province [96]
0
0
Budapest
Query!
Country [97]
0
0
Hungary
Query!
State/province [97]
0
0
Létavértes
Query!
Country [98]
0
0
Hungary
Query!
State/province [98]
0
0
Pécs
Query!
Country [99]
0
0
Hungary
Query!
State/province [99]
0
0
Sopron
Query!
Country [100]
0
0
India
Query!
State/province [100]
0
0
Ahmedabad
Query!
Country [101]
0
0
India
Query!
State/province [101]
0
0
Kolkata
Query!
Country [102]
0
0
India
Query!
State/province [102]
0
0
Secunderabad
Query!
Country [103]
0
0
India
Query!
State/province [103]
0
0
Vellore
Query!
Country [104]
0
0
Indonesia
Query!
State/province [104]
0
0
Jakarta
Query!
Country [105]
0
0
Italy
Query!
State/province [105]
0
0
Bassano Del Grappa (VI)
Query!
Country [106]
0
0
Italy
Query!
State/province [106]
0
0
Firenze
Query!
Country [107]
0
0
Italy
Query!
State/province [107]
0
0
Genova
Query!
Country [108]
0
0
Italy
Query!
State/province [108]
0
0
Milano
Query!
Country [109]
0
0
Italy
Query!
State/province [109]
0
0
Modena
Query!
Country [110]
0
0
Italy
Query!
State/province [110]
0
0
Monza (MB)
Query!
Country [111]
0
0
Italy
Query!
State/province [111]
0
0
Napoli
Query!
Country [112]
0
0
Italy
Query!
State/province [112]
0
0
Rimini
Query!
Country [113]
0
0
Italy
Query!
State/province [113]
0
0
Roma
Query!
Country [114]
0
0
Italy
Query!
State/province [114]
0
0
Torino
Query!
Country [115]
0
0
Italy
Query!
State/province [115]
0
0
Udine
Query!
Country [116]
0
0
Italy
Query!
State/province [116]
0
0
Vittoria (RG)
Query!
Country [117]
0
0
Japan
Query!
State/province [117]
0
0
Kochi
Query!
Country [118]
0
0
Japan
Query!
State/province [118]
0
0
Niigata
Query!
Country [119]
0
0
Japan
Query!
State/province [119]
0
0
Osaka
Query!
Country [120]
0
0
Japan
Query!
State/province [120]
0
0
Tokyo
Query!
Country [121]
0
0
Japan
Query!
State/province [121]
0
0
Chiba
Query!
Country [122]
0
0
Japan
Query!
State/province [122]
0
0
Kobe-shi, Hyogo
Query!
Country [123]
0
0
Korea, Republic of
Query!
State/province [123]
0
0
Gyeongsangnam-do
Query!
Country [124]
0
0
Korea, Republic of
Query!
State/province [124]
0
0
Busan
Query!
Country [125]
0
0
Korea, Republic of
Query!
State/province [125]
0
0
Daejeon
Query!
Country [126]
0
0
Korea, Republic of
Query!
State/province [126]
0
0
Jeonnam
Query!
Country [127]
0
0
Korea, Republic of
Query!
State/province [127]
0
0
Seoul
Query!
Country [128]
0
0
Korea, Republic of
Query!
State/province [128]
0
0
Suwon-si
Query!
Country [129]
0
0
Kuwait
Query!
State/province [129]
0
0
Kuwait
Query!
Country [130]
0
0
Lithuania
Query!
State/province [130]
0
0
Vilnius
Query!
Country [131]
0
0
Malaysia
Query!
State/province [131]
0
0
Jalan Pahang
Query!
Country [132]
0
0
Malaysia
Query!
State/province [132]
0
0
Kuala Lumpur
Query!
Country [133]
0
0
Norway
Query!
State/province [133]
0
0
Bergen
Query!
Country [134]
0
0
Peru
Query!
State/province [134]
0
0
Callao
Query!
Country [135]
0
0
Philippines
Query!
State/province [135]
0
0
Manila
Query!
Country [136]
0
0
Poland
Query!
State/province [136]
0
0
Warszawa
Query!
Country [137]
0
0
Portugal
Query!
State/province [137]
0
0
Lisboa
Query!
Country [138]
0
0
Portugal
Query!
State/province [138]
0
0
Porto
Query!
Country [139]
0
0
Romania
Query!
State/province [139]
0
0
Bucuresti
Query!
Country [140]
0
0
Romania
Query!
State/province [140]
0
0
Cluj-Napoca
Query!
Country [141]
0
0
Russian Federation
Query!
State/province [141]
0
0
Moscow
Query!
Country [142]
0
0
Russian Federation
Query!
State/province [142]
0
0
Nizhny Novgorod
Query!
Country [143]
0
0
Russian Federation
Query!
State/province [143]
0
0
Saint-Peterburg
Query!
Country [144]
0
0
Saudi Arabia
Query!
State/province [144]
0
0
Al Mubarraz
Query!
Country [145]
0
0
Serbia
Query!
State/province [145]
0
0
Novi Sad
Query!
Country [146]
0
0
Singapore
Query!
State/province [146]
0
0
Singapore
Query!
Country [147]
0
0
Slovakia
Query!
State/province [147]
0
0
Bratislava
Query!
Country [148]
0
0
Slovenia
Query!
State/province [148]
0
0
Slovenj Gradec
Query!
Country [149]
0
0
Spain
Query!
State/province [149]
0
0
Barakaldo
Query!
Country [150]
0
0
Spain
Query!
State/province [150]
0
0
Barcelona
Query!
Country [151]
0
0
Spain
Query!
State/province [151]
0
0
La Coruña
Query!
Country [152]
0
0
Spain
Query!
State/province [152]
0
0
Tarragona
Query!
Country [153]
0
0
Spain
Query!
State/province [153]
0
0
Vigo
Query!
Country [154]
0
0
Sweden
Query!
State/province [154]
0
0
Göteborg
Query!
Country [155]
0
0
Sweden
Query!
State/province [155]
0
0
Stockholm
Query!
Country [156]
0
0
Taiwan
Query!
State/province [156]
0
0
Chiayi
Query!
Country [157]
0
0
Taiwan
Query!
State/province [157]
0
0
Hualien
Query!
Country [158]
0
0
Taiwan
Query!
State/province [158]
0
0
Kaohsiung City
Query!
Country [159]
0
0
Taiwan
Query!
State/province [159]
0
0
Kaohsiung Hsien,
Query!
Country [160]
0
0
Taiwan
Query!
State/province [160]
0
0
Kaohsiung
Query!
Country [161]
0
0
Taiwan
Query!
State/province [161]
0
0
New Taipei City
Query!
Country [162]
0
0
Taiwan
Query!
State/province [162]
0
0
Putz City, Chia-yi
Query!
Country [163]
0
0
Taiwan
Query!
State/province [163]
0
0
Taichung City
Query!
Country [164]
0
0
Taiwan
Query!
State/province [164]
0
0
Taichung
Query!
Country [165]
0
0
Taiwan
Query!
State/province [165]
0
0
Tainan
Query!
Country [166]
0
0
Taiwan
Query!
State/province [166]
0
0
Taipei
Query!
Country [167]
0
0
Taiwan
Query!
State/province [167]
0
0
Tao Yuan County
Query!
Country [168]
0
0
Thailand
Query!
State/province [168]
0
0
Bangkok
Query!
Country [169]
0
0
Thailand
Query!
State/province [169]
0
0
Khon Kaen
Query!
Country [170]
0
0
United Kingdom
Query!
State/province [170]
0
0
Cambridgeshire
Query!
Country [171]
0
0
United Kingdom
Query!
State/province [171]
0
0
Belfast
Query!
Country [172]
0
0
United Kingdom
Query!
State/province [172]
0
0
Birmingham
Query!
Country [173]
0
0
United Kingdom
Query!
State/province [173]
0
0
London
Query!
Country [174]
0
0
United Kingdom
Query!
State/province [174]
0
0
Manchester
Query!
Country [175]
0
0
United Kingdom
Query!
State/province [175]
0
0
Salford
Query!
Country [176]
0
0
Vietnam
Query!
State/province [176]
0
0
Hanoi
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Genzyme, a Sanofi Company
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Registry are: * To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; * To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; * To characterize and describe the Fabry population as a whole; * To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. 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. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.
Query!
Trial website
https://clinicaltrials.gov/study/NCT00196742
Query!
Trial related presentations / publications
Germain DP, Brand E, Burlina A, Cecchi F, Garman SC, Kempf J, Laney DA, Linhart A, Marodi L, Nicholls K, Ortiz A, Pieruzzi F, Shankar SP, Waldek S, Wanner C, Jovanovic A. Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study. Mol Genet Genomic Med. 2018 Apr 12;6(4):492-503. doi: 10.1002/mgg3.389. Online ahead of print. Wilcox WR, Feldt-Rasmussen U, Martins AM, Ortiz A, Lemay RM, Jovanovic A, Germain DP, Varas C, Nicholls K, Weidemann F, Hopkin RJ. Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry. JIMD Rep. 2018;38:45-51. doi: 10.1007/8904_2017_28. Epub 2017 May 17. Hopkin RJ, Cabrera G, Charrow J, Lemay R, Martins AM, Mauer M, Ortiz A, Patel MR, Sims K, Waldek S, Warnock DG, Wilcox WR. Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry. Mol Genet Metab. 2016 Sep;119(1-2):151-9. doi: 10.1016/j.ymgme.2016.06.007. Epub 2016 Jun 13. Wanner C, Feldt-Rasmussen U, Ortiz A. Plain language summary of a study looking at heart muscle thickness and kidney function in women with Fabry disease who received agalsidase beta treatment. Future Cardiol. 2022 Sep;18(10):755-763. doi: 10.2217/fca-2022-0047. Epub 2022 Sep 8. Ortiz A, Abiose A, Bichet DG, Cabrera G, Charrow J, Germain DP, Hopkin RJ, Jovanovic A, Linhart A, Maruti SS, Mauer M, Oliveira JP, Patel MR, Politei J, Waldek S, Wanner C, Yoo HW, Warnock DG. Time to treatment benefit for adult patients with Fabry disease receiving agalsidase beta: data from the Fabry Registry. J Med Genet. 2016 Jul;53(7):495-502. doi: 10.1136/jmedgenet-2015-103486. Epub 2016 Mar 18. Germain DP, Charrow J, Desnick RJ, Guffon N, Kempf J, Lachmann RH, Lemay R, Linthorst GE, Packman S, Scott CR, Waldek S, Warnock DG, Weinreb NJ, Wilcox WR. Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease. J Med Genet. 2015 May;52(5):353-8. doi: 10.1136/jmedgenet-2014-102797. Epub 2015 Mar 20.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Study Director
Query!
Address
0
0
Genzyme, a Sanofi Company
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Trial Transparency
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
800-633-1610
Query!
Fax
0
0
Query!
Email
0
0
[email protected]
Query!
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
Download to PDF