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

MPS VI Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01387854
• Other study ID #: ASB-00-03
• Status: Completed
• Phase: N/A
• First received: June 29, 2011
• Last updated: March 31, 2014
• Start date: June 2011
• Est. completion date: February 2013

Study information

• Verified date: March 2014
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Observational

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 59
• Est. completion date: February 2013
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

N/A

Related Conditions & MeSH terms

• MPS VI

Locations

Country	Name	City	State
Australia	Women's and Children's hospital	North Adelaide
Brazil	Hospital de Clinicas de Porto Alegre	Porto Alegre
France	Hôpital Femme Mère Enfant	Lyon
Germany	Prof Michael Beck	Mainz
Portugal	Al. Prof Hernani Monteiro	Porto
United Kingdom	Manchester Academic Health Sciences Centre	Manchester
United States	Children's Hospital and Research Center	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  France,  Germany,  Portugal,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Linear Comparison of assessments completed for Survey Study ASB-00-02 and and Re-Survey StudyASB-00-03	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.	Variable dependent on date of assessment performed fo rthe Survey STudy ASB-00-02	No