Skip to main content
OpenTrials
Completed

NCT Number: NCT01387854

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

MPS VI Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis VI Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Women's and Children's hospital, North Adelaide, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

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.

Treatment and study plan

Primary outcomes

  1. Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03

    Time frame: Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02

    This study is designed to be performed in a similar manner to the Survey Study ASB-00-02. Similar assessments will be performed in order to make linear comparisons in outcomes over a long-term period.

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

A Re-Survey Study of Patients With MPS VI (Maroteaux-Lamy Syndrome) Who Previously Participated in ASB-00-02

Important dates

Study start
2011
Primary completion
2012
Study completion
2013
First posted
Jul 6, 2011
Registry last updated
Apr 2, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.