Natural History Observational Study of MPS IIIa in SMC

Samsung Medical Center

Status: Not yet enrolling

Conditions: Sanfilippo Syndrome A

Treatments: Other: assessment

Study type: Observational

Funder types: Other

Identifiers

NCT04918641

SMC-2020-10-164-003

Details and patient eligibility

About

To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identify potential endpoints for future treatment trials.

Enrollment: 25 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of MPS IIIA
• Has the ability to comply with protocol requirements, in the opinion of the investigator

Exclusion criteria

• Patient's assent is unattainable, or the patient's parent(s), or patient's legally authorized representative(s) is/are unable to understand the nature, scope, and possible consequences of the study, or do/does not agree to comply with the protocol defined schedule of assessments.

Trial design

25 participants in 1 patient group

No treatment

Description:

This is a longitudinal, prospective, observational, natural history study of patients with MPS IIIA to identify potential surrogate endpoints for future trials via standardized clinical, biochemical, neurocognitive, developmental, behavioral, and imaging measures.

Treatment:

Other: assessment