Natural History Observational Study of MPS IIIa in SMC

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

25 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-06-30

Study Completion Date

2024-07-28

Brief Summary

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

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Detailed Description

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Conditions

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Sanfilippo Syndrome A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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No treatment

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.

assessment

Intervention Type OTHER

Physical, developmental, neurological, behavioral, and neurocognitive assessments

Interventions

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assessment

Physical, developmental, neurological, behavioral, and neurocognitive assessments

Intervention Type OTHER

Eligibility Criteria

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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.

Eligible Sex

ALL

Accepts Healthy Volunteers

No