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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00214773




Registration number
NCT00214773
Ethics application status
Date submitted
13/09/2005
Date registered
22/09/2005
Date last updated
5/04/2021

Titles & IDs
Public title
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Scientific title
MPS VI Clinical Surveillance Program (CSP)
Secondary ID [1] 0 0
MPSVI CSP
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) 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
Observational - No intervention. This is an observational program.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase.
Timepoint [1] 0 0
at least 15 years

Eligibility
Key inclusion criteria
Inclusion Criteria

All patients must meet the following criteria to qualify for enrollment in the CSP:

* Patient or patient's parent or legal guardian, if child is under 18 year old or is unable to consent, has provided a signed Patient Information and Authorization Form.
* Patient has laboratory results confirming a diagnosis of MPS VI disease based on detection of deficient ARSB activity (on fibroblasts, leucocytes or dried blood spots)and/or abnormality on the ARSB gene.
* Patient is willing to undergo general assessments to establish baseline data or permits physician to enter assessment data recorded prior to CSP entry if available in the patient's medical records. General assessments include: urinary GAG level, urinary protein level, serum sample for antibody levels, height, weight, and patient history.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/07/2005
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2020
Sample size
Target
Accrual to date
Final
237
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Westmead Hospital - Wentworthville
Recruitment hospital [2] 0 0
Royal Children's Hospital, Brisbane - Brisbane
Recruitment hospital [3] 0 0
Women and Children's Hospital - Adelaide
Recruitment hospital [4] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [5] 0 0
Princess Margaret Hospital for Children - Subiaco
Recruitment postcode(s) [1] 0 0
NSW2145 - Wentworthville
Recruitment postcode(s) [2] 0 0
4029 - Brisbane
Recruitment postcode(s) [3] 0 0
5006 - Adelaide
Recruitment postcode(s) [4] 0 0
- Parkville
Recruitment postcode(s) [5] 0 0
6008 - Subiaco
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Louisiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New Jersey
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
North Carolina
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
South Dakota
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Austria
State/province [18] 0 0
Steiermark
Country [19] 0 0
Belgium
State/province [19] 0 0
Wallonie
Country [20] 0 0
France
State/province [20] 0 0
Rhône-alpes
Country [21] 0 0
France
State/province [21] 0 0
Paris
Country [22] 0 0
Germany
State/province [22] 0 0
Mecklenburg-vorpommern
Country [23] 0 0
Germany
State/province [23] 0 0
Niedersachsen
Country [24] 0 0
Germany
State/province [24] 0 0
Rheinland-Pfalz RP
Country [25] 0 0
Germany
State/province [25] 0 0
Berlin
Country [26] 0 0
Germany
State/province [26] 0 0
Hamburg
Country [27] 0 0
Ireland
State/province [27] 0 0
Limerick
Country [28] 0 0
Italy
State/province [28] 0 0
Catania
Country [29] 0 0
Italy
State/province [29] 0 0
Veneto
Country [30] 0 0
Italy
State/province [30] 0 0
Monza
Country [31] 0 0
Lithuania
State/province [31] 0 0
Vilnius
Country [32] 0 0
Netherlands
State/province [32] 0 0
Rotterdam
Country [33] 0 0
Portugal
State/province [33] 0 0
Porto
Country [34] 0 0
Sweden
State/province [34] 0 0
Stockholm
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Birmingham
Country [36] 0 0
United Kingdom
State/province [36] 0 0
London
Country [37] 0 0
United Kingdom
State/province [37] 0 0
Manchester
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
Brief summary
The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further characterize the long-term safety of Naglazyme® treatment.
Trial website
https://clinicaltrials.gov/study/NCT00214773
Trial related presentations / publications
Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR; CSP Study Group. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Mol Genet Metab. 2016 Aug;118(4):310-8. doi: 10.1016/j.ymgme.2016.06.001. Epub 2016 Jun 3.
Public notes

Contacts
Principal investigator
Name 0 0
Julie Johnson
Address 0 0
BioMarin Pharmaceutical
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/NCT00214773