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

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Trial Status Completed

Trial Identifier

NCT01387854

Condition

MPS VI

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

59

Study Started:

2011-06

Study Updated:

2014-04-02

Trial Locations

  • Children's Hospital and Research Center

    Oakland, California, United States

  • Women's and Children's hospital

    North Adelaide, , Australia

  • Hospital de Clinicas de Porto Alegre

    Porto Alegre, , Brazil

  • Hôpital Femme Mère Enfant

    Lyon, , France

  • Prof Michael Beck

    Mainz, , Germany

  • Al. Prof Hernani Monteiro

    Porto, , Portugal

  • Manchester Academic Health Sciences Centre

    Manchester, , United Kingdom

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

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