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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00231400




Registration number
NCT00231400
Ethics application status
Date submitted
30/09/2005
Date registered
4/10/2005
Date last updated
20/11/2024

Titles & IDs
Public title
Pompe Disease Registry Protocol
Scientific title
Pompe Disease Registry
Secondary ID [1] 0 0
DIREGC07005
Secondary ID [2] 0 0
DIREGC07005
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Glycogen Storage Disease Type II 0 0
Pompe Disease 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
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease
Timepoint [1] 0 0
maximum 30 years

Eligibility
Key inclusion criteria
All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
There are no exclusion criteria in this 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
15/09/2004
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/01/2034
Actual
Sample size
Target
2000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,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 : 154087 - Brisbane
Recruitment hospital [4] 0 0
Investigational Site Number : 152096 - Brisbane
Recruitment hospital [5] 0 0
Investigational Site Number : 151002 - Adelaide
Recruitment hospital [6] 0 0
Investigational Site Number : 151015 - Murdoch
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4029 - Brisbane
Recruitment postcode(s) [3] 0 0
4101 - Brisbane
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
6150 - Murdoch
Recruitment outside Australia
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United States of America
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Alabama
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United States of America
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Arizona
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Arkansas
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United States of America
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California
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Colorado
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United States of America
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Connecticut
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District of Columbia
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United States of America
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Florida
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United States of America
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Georgia
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United States of America
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Illinois
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Indiana
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Iowa
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Kansas
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Kentucky
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United States of America
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Louisiana
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United States of America
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Maine
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United States of America
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Maryland
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Massachusetts
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Michigan
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Minnesota
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Missouri
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Nebraska
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Nevada
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New Jersey
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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Rhode Island
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South Carolina
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Tennessee
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Texas
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Utah
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Virginia
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Washington
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United States of America
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Wisconsin
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Argentina
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Buenos Aires
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Belgium
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Antwerp
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Belgium
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Bruges
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Belgium
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Brussels
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Belgium
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Bruxelles
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Belgium
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Edegem
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Belgium
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Gent
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Belgium
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Gosselies
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Belgium
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Leuven
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Brazil
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Ceará
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Brazil
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Mato Grosso
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Brazil
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Rio Grande Do Sul
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Brazil
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São Paulo
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Brazil
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Rio de Janeiro
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Bulgaria
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Sofia
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Alberta
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Canada
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British Columbia
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Canada
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Manitoba
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Canada
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New Brunswick
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Canada
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Ontario
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Canada
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Quebec
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Chile
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Punta Arenas
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Chile
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Santiago
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China
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Beijing
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China
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Guangzhou
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China
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Jinan
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China
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Shanghai
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Colombia
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Bogota
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Croatia
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Zagreb
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Czechia
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Praha 2
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Denmark
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København Ø
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Bordeaux
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France
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Brest
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BRON Cedex
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Caen
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France
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La Tronche
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Lille
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France
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Marseille Cedex 05
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France
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Nancy
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France
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Nantes
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France
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Nice Cedex 1
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France
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Nîmes
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France
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Paris
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France
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St-priest-en-jarez
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France
State/province [82] 0 0
Strasbourg
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France
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Toulouse
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France
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Tours
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France
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Vannes Cedex
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Germany
State/province [86] 0 0
Berlin
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Germany
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Bochum
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Germany
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Bonn
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Germany
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Freiburg
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Germany
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Gießen
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Germany
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Halle (Saale)
Country [92] 0 0
Germany
State/province [92] 0 0
Hamburg
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Germany
State/province [93] 0 0
Mainz
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Germany
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München
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Germany
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Münster
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Greece
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Thessaloniki
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Hong Kong
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Kowloon
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Hungary
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Debrecen
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Hungary
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Nyíregyháza
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Hungary
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Pã©cs
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Hungary
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Székesfehérvár
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India
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Ahmedabad
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India
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Kolkata
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India
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Secunderabad
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India
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Vellore
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Indonesia
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Jakarta
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Israel
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Beer Sheva
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Israel
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Haifa
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Israel
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Tel Aviv
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Italy
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Brescia
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Italy
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Cagliari
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Italy
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Ferrara
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Italy
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Firenze
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Italy
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Genova
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Italy
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Messina
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Italy
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Milano
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Italy
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Milan
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Italy
State/province [118] 0 0
Monza
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Italy
State/province [119] 0 0
Napoli
Country [120] 0 0
Italy
State/province [120] 0 0
Padova
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Italy
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Padua
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Italy
State/province [122] 0 0
Pavia
Country [123] 0 0
Italy
State/province [123] 0 0
Roma
Country [124] 0 0
Italy
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Torino
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Italy
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Udine
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Japan
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Kyoto
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Japan
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Okinawa
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Jordan
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Amman
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Korea, Republic of
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Gyeonggi-do
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Korea, Republic of
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Gyeongsangnam-do
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Korea, Republic of
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Seoul-teukbyeolsi
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Korea, Republic of
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Daejeon
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Korea, Republic of
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Jeonnam
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Korea, Republic of
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Seoul
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Kuwait
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Kuwait
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Lebanon
State/province [136] 0 0
Beirut
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Malaysia
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Jalan Pahang
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Malaysia
State/province [138] 0 0
Kuala Lumpur
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Netherlands
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Rotterdam
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Pakistan
State/province [140] 0 0
Karachi
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Pakistan
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Lahore
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Philippines
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Manila
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Poland
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Warszawa
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Portugal
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Coimbra
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Portugal
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Lisboa
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Romania
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Bucuresti
Country [147] 0 0
Russian Federation
State/province [147] 0 0
Moscow
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Saudi Arabia
State/province [148] 0 0
Makkah
Country [149] 0 0
Saudi Arabia
State/province [149] 0 0
Qatif
Country [150] 0 0
Serbia
State/province [150] 0 0
Belgrade
Country [151] 0 0
Singapore
State/province [151] 0 0
Singapore
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Slovakia
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Bratislava
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Taiwan
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Chiayi
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Taiwan
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Hualien
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Taiwan
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Kaohsiung City
Country [156] 0 0
Taiwan
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Kaohsiung
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Taiwan
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New Taipei City
Country [158] 0 0
Taiwan
State/province [158] 0 0
Putz City, Chia-yi
Country [159] 0 0
Taiwan
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Taichung
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Taiwan
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Tainan City
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Taiwan
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Taipei City
Country [162] 0 0
Taiwan
State/province [162] 0 0
Taipei
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Taiwan
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Taoyuan City
Country [164] 0 0
Thailand
State/province [164] 0 0
Bangkok
Country [165] 0 0
Thailand
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Khon Kaen
Country [166] 0 0
United Kingdom
State/province [166] 0 0
Cambridgeshire
Country [167] 0 0
United Kingdom
State/province [167] 0 0
Birmingham
Country [168] 0 0
United Kingdom
State/province [168] 0 0
Cardiff
Country [169] 0 0
United Kingdom
State/province [169] 0 0
Glasgow
Country [170] 0 0
United Kingdom
State/province [170] 0 0
London
Country [171] 0 0
United Kingdom
State/province [171] 0 0
Manchester
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United Kingdom
State/province [172] 0 0
Newcastle
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United Kingdom
State/province [173] 0 0
Salford
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Vietnam
State/province [174] 0 0
Hanoi
Country [175] 0 0
Vietnam
State/province [175] 0 0
Ho Chi Minh City

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 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

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/NCT00231400