Trials / Unknown
UnknownNCT04918641
Natural History Observational Study of MPS IIIa in SMC
Prospective, Natural History Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
- Status
- Unknown
- Phase
- —
- Study type
- Observational
- Enrollment
- 25 (estimated)
- Sponsor
- Samsung Medical Center · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identify potential endpoints for future treatment trials.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | assessment | Physical, developmental, neurological, behavioral, and neurocognitive assessments |
Timeline
- Start date
- 2021-06-30
- Primary completion
- 2023-07-28
- Completion
- 2024-07-28
- First posted
- 2021-06-09
- Last updated
- 2021-06-09
Source: ClinicalTrials.gov record NCT04918641. Inclusion in this directory is not an endorsement.