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.
This is a multicenter, multinational, longitudinal, observational study in children aged up to and including 9 years, who have been diagnosed with MPS IIIA. The study will detail the natural course of MPS IIIA via standardized clinical, biochemical, neurocognitive, developmental, behavioral and imaging measures. This information is anticipated to inform the design and interpretation of future interventional studies.
Study Type
OBSERVATIONAL
Enrollment
23
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil
Armand Trousseau Public Hospital
Paris, France
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
Academic Medical Center, Emma Children's Hospital
Amsterdam, Netherlands
The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition
Time frame: Baseline, and every 6 months, for up to 24 months
Change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior scale
Time frame: Baseline and every 6 months up to 24 months
Sleep disturbances measured by Actigraphy
Time frame: Baseline and every 3 months up to 24 months
Patient Quality of Life Questionnaires
Time frame: Baseline and every 6 months up to 24 months
Change from baseline in total cortical grey matter volume
Time frame: Baseline, 12 months, 24 months
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Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom