UnknownNot Applicable

Natural History Observational Study of MPS IIIa in SMC

25 participantsStarted 2021-06-30

Plain-language summary

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

Who can participate

SexALL

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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.

What they're measuring

Change From Baseline in Bayley Scales of Infant Development-III/Kaufman Assessment Battery for Children-II (BSID-III/KABC-II) Age-Equivalent Scores

Timeframe: Baseline, 12 months, and 24 months

Change From Baseline in Vineland Adaptive Behavior Scales-II (VABS-II) Age-equivalent Scores

Timeframe: Baseline, 12 months, and 24 months

Change from Baseline in MPS health Assessment Questionnaire, Sanfilippo Behavior Rating Scale

Timeframe: Baseline, 12 months, and 24 months

Trial details

NCT IDNCT04918641

SponsorSamsung Medical Center

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2023-07-28

Contact for this trial

SungYoon Cho

+82-2-3410-3537 nadri1217@naver.com

View on ClinicalTrials.gov More Sanfilippo Syndrome A trials