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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01387854




Registration number
NCT01387854
Ethics application status
Date submitted
29/06/2011
Date registered
6/07/2011
Date last updated
2/04/2014

Titles & IDs
Public title
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
Scientific title
A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02
Secondary ID [1] 0 0
ASB-00-03
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
MPS VI 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
Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03
Timepoint [1] 0 0
Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02

Eligibility
Key inclusion criteria
* Willing and able to provide written, signed informed consent, or in the case of patients under the age of 16 years, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
* Previously participated in Survey Study ASB-00-02, and met the criteria of a MPS VI diagnosis.
* Willing to perform all study assessments and procedures as physically possible.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Concurrent disease or condition that would interfere with study participation or safety.
* Any condition that, in the view of the Investigator would place the patient at high risk of not completing the study.
* Concurrent enrollment and randomization into a clinical study of MPS VI treatment. Patients enrolled in the BioMarin MPS VI Clinical Surveillance Program may be enrolled in the Re-survey Study ASB-00-03.

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/02/2013
Sample size
Target
Accrual to date
Final
59
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Women's and Children's hospital - North Adelaide
Recruitment postcode(s) [1] 0 0
- North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
Brazil
State/province [2] 0 0
Porto Alegre
Country [3] 0 0
France
State/province [3] 0 0
Lyon
Country [4] 0 0
Germany
State/province [4] 0 0
Mainz
Country [5] 0 0
Portugal
State/province [5] 0 0
Porto
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
There is limited information on the long-term effects of treating patients with MPS VI with Naglazyme® and limited data on the natural history of treated and untreated MPS VI patients. The Re-survey Study ASB-00-03 will assist in understanding the effects of long-term Naglazyme treatment and the natural history of the MPS VI patient population.
Trial website
https://clinicaltrials.gov/study/NCT01387854
Trial related presentations / publications
Giugliani R, Lampe C, Guffon N, Ketteridge D, Leao-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01387854