Trial record 13 of 38 for:
Open Studies | "Mucopolysaccharidoses"
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
Verified September 2014 by BioMarin Pharmaceutical
Information provided by (Responsible Party):
First received: September 13, 2005
Last updated: September 8, 2014
Last verified: September 2014
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.
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
||Observational Model: Cohort
Time Perspective: Prospective
||MPS VI Clinical Surveillance Program (CSP)
Biospecimen Retention: Samples Without DNA
Primary Outcome Measures:
- 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 ] [ Designated as safety issue: Yes ]
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||July 2020 (Final data collection date for primary outcome measure)
No intervention. This is an observational program.
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
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.
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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00214773
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||September 13, 2005
||September 8, 2014
||United States: Food and Drug Administration
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on September 22, 2014
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases