Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| The change from baseline in cognitive function using the Bayley scales of infant and toddler development third edition | Baseline, and every 6 months, for up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in the adaptive behavior composite standard score as measured by the Vineland Adaptive Behavior scale | Baseline and every 6 months up to 24 months | |
| Sleep disturbances measured by Actigraphy | Baseline and every 3 months up to 24 months |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Subjects diagnosed with MPS IIIA
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital de Clinicas de Porto Alegre | Porto Alegre | 90035-003 | Brazil | |||
| Armand Trousseau Public Hospital |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Cerebral Spinal Fluid
| Patient Quality of Life Questionnaires | Baseline and every 6 months up to 24 months |
| Change from baseline in total cortical grey matter volume | Baseline, 12 months, 24 months |
| Paris |
| France |
| University Medical Center Hamburg-Eppendorf | Hamburg | Germany |
| Academic Medical Center, Emma Children's Hospital | Amsterdam | Netherlands |
| Great Ormond Street Hospital NHS Foundation Trust | London | United Kingdom |