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Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

This study is currently recruiting participants.
Verified April 2017 by BioMarin Pharmaceutical
Sponsor:
ClinicalTrials.gov Identifier:
NCT00214773
First Posted: September 22, 2005
Last Update Posted: April 21, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
BioMarin Pharmaceutical
September 13, 2005
September 22, 2005
April 21, 2017
July 2005
July 2020   (Final data collection date for primary outcome measure)
To further characterize the natural progression of MPS VI disease, irrespective of treatment modality and to evaluate efficacy and safety treatment with Galsulfase. [ Time Frame: at least 15 years ]
Not Provided
Complete list of historical versions of study NCT00214773 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
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Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
MPS VI Clinical Surveillance Program (CSP)
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.
Not Provided
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   Samples Without DNA
Description:
Urine and serum
Non-Probability Sample
All patients with a confirmed diagnosis of MPS VI disease may participate in the CSP. It is not a requirement that the patients enrolled in the CSP receive Galsulfase 1mg/kg to participate as this is an observational program.
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Not Provided
Observational
No intervention. This is an observational program.
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
200
December 2020
July 2020   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact: MEDINFO MEDINFO (615) 523-0310 MEDINFO@bmrn.com
Australia,   Austria,   Belgium,   France,   Germany,   Ireland,   Italy,   Lithuania,   Netherlands,   Portugal,   Sweden,   United Kingdom,   United States
 
 
NCT00214773
MPSVI CSP
No
Not Provided
Plan to Share IPD: No
BioMarin Pharmaceutical
BioMarin Pharmaceutical
Not Provided
Not Provided
BioMarin Pharmaceutical
April 2017