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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00144794




Registration number
NCT00144794
Ethics application status
Date submitted
2/09/2005
Date registered
5/09/2005
Date last updated
20/11/2024

Titles & IDs
Public title
Mucopolysaccharidosis I (MPS I) Registry
Scientific title
Mucopolysaccharidosis I (MPS I) Registry
Secondary ID [1] 0 0
U1111-1294-8266
Secondary ID [2] 0 0
DIREGC07008
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis I (MPS I) 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
Other metabolic disorders

Intervention/exposure
Study type
Observational
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
To evaluate the long-term effectiveness of Aldurazyme
Timepoint [1] 0 0
Approximately 17 Years

Eligibility
Key inclusion criteria
* All patients with a confirmed diagnosis of MPS I are eligible for inclusion. Confirmed diagnosis is defined as: A. documented biochemical evidence of a deficiency in alpha (a)-L-iduronidase enzyme activity and/or B. mutation(s) in the gene coding for a-L-iduronidase, or measurable clinical signs and symptoms of MPS I
* For all patients there should be a completed patient authorization form
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.

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
20/11/2003
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/01/2034
Actual
Sample size
Target
1500
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD
Recruitment hospital [1] 0 0
Investigational Site Number : 151069 - Westmead
Recruitment hospital [2] 0 0
Investigational Site Number : 152096 - Brisbane
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4101 - Brisbane
Recruitment outside Australia
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United States of America
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Arizona
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United States of America
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Arkansas
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California
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Colorado
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Connecticut
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District of Columbia
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Florida
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Georgia
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Illinois
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Indiana
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Iowa
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Kentucky
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Louisiana
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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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Oregon
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Pennsylvania
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South Carolina
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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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Argentina
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Belgium
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Brussels
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Leuven
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Belgium
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Roeselare
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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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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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Los Angeles
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Chile
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Talcahuano
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Colombia
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Bogotá
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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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Egypt
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Cairo
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France
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BRON Cedex
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France
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Marseille Cedex 05
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France
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Paris Cedex 12
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France
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Paris
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France
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Vandœuvre-lès-nancy
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Hamburg
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Germany
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Mainz
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Kowloon
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Hungary
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Pécs
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India
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Ahmedabad
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India
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Kolkata
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Secunderabad
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Vellore
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Jakarta
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Moscow
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Qatif
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Singapore
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Stockholm
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Taiwan
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Chiayi
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Hualien
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Taiwan
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Kaohsiung City
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Taiwan
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Kaohsiung
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Taiwan
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New Taipei City
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Taiwan
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Putz City, Chia-yi
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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
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Taiwan
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Taipei
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Taiwan
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Taoyuan City
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Thailand
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Bangkok
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Thailand
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Khon Kaen
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United Kingdom
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Birmingham
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United Kingdom
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Glasgow
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United Kingdom
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London
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United Kingdom
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Manchester
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United Kingdom
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Salford
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Vietnam
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Hanoi
Country [122] 0 0
Vietnam
State/province [122] 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
Medical Monitor
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
For site information, send an email with site number to
Address 0 0
Country 0 0
Phone 0 0
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/NCT00144794