Natural History Study of Patients With MPS IIIA
NCT02746341 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 23
Last updated 2021-08-31
Summary
Evaluate the clinical progression in patients with MPS IIIA who are untreated with any investigational product and to obtain standardized assessments: neurocognitive, behavioral, sleep-wake habits and effect of MPS IIIA on the quality of life of patients and their families.
Conditions
- Mucopolysaccharidosis IIIA
Sponsors & Collaborators
-
LYSOGENE
lead INDUSTRY
Eligibility
- Min Age
- 0 Years
- Max Age
- 9 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2016-04-30
- Primary Completion
- 2019-05-31
- Completion
- 2019-05-31
Countries
- Brazil
- France
- Germany
- Netherlands
- United Kingdom
Study Locations
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