Trials / Completed
CompletedNCT02746341
Natural History Study of Patients With MPS IIIA
An Observational, Prospective, Multi-center, Natural History Study of Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 23 (actual)
- Sponsor
- LYSOGENE · Industry
- Sex
- All
- Age
- 0 Years – 9 Years
- Healthy volunteers
- Not accepted
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.
Detailed description
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.
Conditions
Timeline
- Start date
- 2016-04-01
- Primary completion
- 2019-05-01
- Completion
- 2019-05-01
- First posted
- 2016-04-21
- Last updated
- 2021-08-31
Locations
5 sites across 5 countries: Brazil, France, Germany, Netherlands, United Kingdom
Source: ClinicalTrials.gov record NCT02746341. Inclusion in this directory is not an endorsement.