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

Stopped early

Conditions studied: MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome

In brief

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.

Key facts

Study ID

NCT00787995

Run by

BioMarin Pharmaceutical

People needed

353

Starts

2008-10-01

Expected to finish

2014-07-01

Last updated by the study team

2014-12-08

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 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.

You may not qualify if…

• 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.

Where it is running

• Study site — Oakland, California, United States
• Study site — Chicago, Illinois, United States
• Study site — Córdoba, Argentina
• Study site — Porto Alegre, Brazil
• Study site — São Paulo, Brazil
• Study site — Montreal, Quebec, Canada
• Study site — Lyon, France
• Study site — Paris, France
• Study site — Mainz, Germany
• Study site — Monza, Italy
• Study site — Amsterdam, Netherlands
• Study site — Taipei, Taiwan
• Study site — Birmingham, United Kingdom
• Study site — London, United Kingdom
• Study site — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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