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

NCT01387854 | Completed

• 1 other identifier: ASB-00-03
• Study Type: observational
• Target: 59
• Locations: 7 countries
• Sites: 7

Brief 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.

Conditions

MPS VI

Outcome Measures

Primary Outcomes (1)

• 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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with MPS VI who previously participated in ASB-00-02

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.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (7)

Children's Hospital and Research Center

Oakland, California, 94609, United States

Location

Women's and Children's hospital

North Adelaide, Australia

Location

Hospital de Clinicas de Porto Alegre

Porto Alegre, 21615, Brazil

Location

Hôpital Femme Mère Enfant

Lyon, Lyon, 69677, France

Location

Prof Michael Beck

Mainz, Mainz, 55131, Germany

Location

Al. Prof Hernani Monteiro

Porto, Porto District, 4202-451, Portugal

Location

Manchester Academic Health Sciences Centre

Manchester, Manchester, M13 9 WL, United Kingdom

Location

Related Publications (1)

• Giugliani R, Lampe C, Guffon N, Ketteridge D, Leao-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.

  PMID: 24764221 DERIVED

MeSH Terms

Conditions

Mucopolysaccharidosis VI

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 29, 2011
• First Posted: July 6, 2011
• Study Start: June 1, 2011
• Primary Completion: October 1, 2012
• Study Completion: February 1, 2013
• Last Updated: April 2, 2014

Record last verified: 2014-03

Locations

BR (1); DE (1); AU (1); FR (1); GB (1); US (1); PT (1)