A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
Status: | Recruiting |
---|---|
Conditions: | Orthopedic, Metabolic |
Therapuetic Areas: | Pharmacology / Toxicology, Orthopedics / Podiatry |
Healthy: | No |
Age Range: | Any |
Updated: | 2/7/2015 |
Start Date: | October 2008 |
Contact: | Kenny Jones |
Phone: | 866-961-8212 |
A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
This multicenter, multinational, longitudinal study in subjects diagnosed with MPS IVA will
better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA
disease.
better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA
disease.
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.
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