Status:
TERMINATED
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
MPS IV A
Mucopolysaccharidosis IVA
Eligibility:
All Genders
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 bioche...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 1 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
July 1 2014
Estimated Enrollment :
353 Patients enrolled
Trial Details
Trial ID
NCT00787995
Start Date
October 1 2008
End Date
July 1 2014
Last Update
December 8 2014
Active Locations (15)
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1
Oakland, California, United States
2
Chicago, Illinois, United States
3
Córdoba, Argentina
4
Porto Alegre, Brazil