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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
10/06/2024
Titles & IDs
Public title
Mucopolysaccharidosis I (MPS I) Registry
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Scientific title
Mucopolysaccharidosis I (MPS I) Registry
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Secondary ID [1]
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U1111-1294-8266
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Secondary ID [2]
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DIREGC07008
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis I (MPS I)
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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0
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Other metabolic disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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To evaluate the long-term effectiveness of Aldurazyme
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Assessment method [1]
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Timepoint [1]
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Approximately 17 Years
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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
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* No exclusion criteria for participation in the MPS I Registry. NOTE: Registry participation does not exclude participation in other clinical studies.
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Study design
Purpose
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Recruitment
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Recruiting
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Data analysis
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
20/11/2003
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Date of last data collection
Anticipated
31/01/2034
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Actual
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Sample size
Target
1500
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,QLD
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Recruitment hospital [1]
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Investigational Site Number : 151069 - Westmead
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Recruitment hospital [2]
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Investigational Site Number : 152096 - Brisbane
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2145 - Westmead
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4101 - Brisbane
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Name
Genzyme, a Sanofi Company
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Ethics approval
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Summary
Brief summary
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities. The objectives of the Registry are: * To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase) * To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I * To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care
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Trial website
https://clinicaltrials.gov/study/NCT00144794
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Trial related presentations / publications
Viskochil D, Muenzer J, Guffon N, Garin C, Munoz-Rojas MV, Moy KA, Hutchinson DT. Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort study. Dev Med Child Neurol. 2017 Dec;59(12):1269-1275. doi: 10.1111/dmcn.13545. Epub 2017 Sep 11. Arn P, Bruce IA, Wraith JE, Travers H, Fallet S. Airway-related symptoms and surgeries in patients with mucopolysaccharidosis I. Ann Otol Rhinol Laryngol. 2015 Mar;124(3):198-205. doi: 10.1177/0003489414550154. Epub 2014 Sep 11. Beck M, Arn P, Giugliani R, Muenzer J, Okuyama T, Taylor J, Fallet S. The natural history of MPS I: global perspectives from the MPS I Registry. Genet Med. 2014 Oct;16(10):759-65. doi: 10.1038/gim.2014.25. Epub 2014 Mar 27.
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Public notes
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Contacts
Principal investigator
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Medical Monitor
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Address
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Genzyme, a Sanofi Company
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For site information, send an email with site number to
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[email protected]
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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
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