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

Completed

Conditions studied: MPS VI

In brief

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.

Key facts

Study ID

NCT01387854

Run by

BioMarin Pharmaceutical

People needed

59

Starts

2011-06-01

Expected to finish

2013-02-01

Last updated by the study team

2014-04-02

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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, Lyon, France
• Prof Michael Beck — Mainz, Mainz, Germany
• Al. Prof Hernani Monteiro — Porto, Porto District, Portugal
• Manchester Academic Health Sciences Centre — Manchester, Manchester, United Kingdom

Full record on ClinicalTrials.gov

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