Status:

UNKNOWN

Natural History Observational Study of MPS IIIa in SMC

Lead Sponsor:

Samsung Medical Center

Conditions:

Sanfilippo Syndrome A

Eligibility:

All Genders

Brief Summary

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

Eligibility Criteria

Inclusion

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

Exclusion

  • 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.

Key Trial Info

Start Date :

June 30 2021

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

July 28 2024

Estimated Enrollment :

25 Patients enrolled

Trial Details

Trial ID

NCT04918641

Start Date

June 30 2021

End Date

July 28 2024

Last Update

June 9 2021

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Natural History Observational Study of MPS IIIa in SMC | DecenTrialz