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

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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