A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Terminated

• Conditions: MPS IV A; Mucopolysaccharidosis IVA; Morquio A Syndrome

• Registration Number: NCT00787995
• Lead Sponsor: BioMarin Pharmaceutical

Brief Summary

This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-10
• Study First Submitted: 2008-11-06
• Study First Submitted QC: 2008-11-06
• Study First Posted: 2008-11-10
• Primary Completion: 2014-07
• Study Completion: 2014-07
• Status Verified: 2014-12
• Last Update Submitted: 2014-12-05
• Last Update Posted: 2014-12-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 353

Inclusion Criteria

• Willing and able to provide written, signed informed consent, or in the case of subjects age < 18 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.
• Documented history of reduced GALNS activity relative to the normal range of the laboratory performing the assay, or documented result of molecular genetic testing confirming diagnosis of MPS IVA.
• Willing and able to comply with all study procedures.

Exclusion Criteria

• Use of any investigational product or investigational medical device within 30 days prior to screening.
• Previous hematopoietic stem cell transplant (HSCT).
• Concurrent disease or condition that would interfere with study participation or pose a safety concern.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Respiratory Function	Study Visit
Endurance	Study Visit