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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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
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
Country [1] 0 0
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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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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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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United States of America
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Kansas
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United States of America
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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
State/province [16] 0 0
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 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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Wisconsin
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Antwerp
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Belgium
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Edegem
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Gent
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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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Bogota
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Zagreb
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Praha 2
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København Ø
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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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France
State/province [77] 0 0
Nantes
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France
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Nice Cedex 1
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France
State/province [79] 0 0
Nîmes
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France
State/province [80] 0 0
Paris
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France
State/province [81] 0 0
St-priest-en-jarez
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France
State/province [82] 0 0
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France
State/province [83] 0 0
Toulouse
Country [84] 0 0
France
State/province [84] 0 0
Tours
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France
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Germany
State/province [87] 0 0
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Germany
State/province [88] 0 0
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Germany
State/province [89] 0 0
Freiburg
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Germany
State/province [90] 0 0
Gießen
Country [91] 0 0
Germany
State/province [91] 0 0
Halle (Saale)
Country [92] 0 0
Germany
State/province [92] 0 0
Hamburg
Country [93] 0 0
Germany
State/province [93] 0 0
Mainz
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Germany
State/province [94] 0 0
München
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Germany
State/province [95] 0 0
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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Roma
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Italy
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Korea, Republic of
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Korea, Republic of
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Karachi
Country [141] 0 0
Pakistan
State/province [141] 0 0
Lahore
Country [142] 0 0
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Manila
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Portugal
State/province [144] 0 0
Coimbra
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Portugal
State/province [145] 0 0
Lisboa
Country [146] 0 0
Romania
State/province [146] 0 0
Bucuresti
Country [147] 0 0
Russian Federation
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Moscow
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Makkah
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Qatif
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Singapore
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Hualien
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Taiwan
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New Taipei City
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Taiwan
State/province [158] 0 0
Putz City, Chia-yi
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Taiwan
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Taichung
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Taiwan
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Taiwan
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Taipei
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Taiwan
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Bangkok
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State/province [166] 0 0
Cambridgeshire
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State/province [167] 0 0
Birmingham
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State/province [168] 0 0
Cardiff
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State/province [169] 0 0
Glasgow
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London
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United Kingdom
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Manchester
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Newcastle
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Salford
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Hanoi
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Vietnam
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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.