A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) (NCT01047306) | Clinical Trial Compass
CompletedNot Applicable
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
United States25 participantsStarted 2010-02-15
Plain-language summary
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including standardized clinical, biochemical, neurocognitive, behavioral, developmental, and imaging measures.
Who can participate
Age range
1 Year
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
.Patient is greater than or equal to 1 year of age and developmental age greater than or equal to 1 year.
Exclusion criteria
. Patient has significant non-MPS IIIA-related CNS impairment or behavioral disturbances, which would confound the scientific integrity or interpretation of study assessments, as determined by the investigator.
. Patients who, for MPS IIIA behavioral-related reasons,in the opinion of the investigator, would preclude performance of study neurocognitive and developmental testing procedures.
. Patients who are pregnant, breast feeding, or female patients of childbearing potential, who will not or cannot comply with the use of an acceptable method of birth control such as condoms, barrier method, oral contraception, etc.
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
1
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, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)
2
Change From Baseline in BSID-III/KABC-II Developmental Quotient (DQ) Scores
Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)
3
Change From Baseline in Vineland Adaptive Behavior Scales-II (VABS-II) Age-equivalent Scores
Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)
4
Change From Baseline in VABS-II Overall DQ Scores
Timeframe: Baseline, 6 months, 12 months, and End of Study (Month 24 assessment or early termination)
. Patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to airway compromise or other conditions.
. Patient or patient family history of neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns.
. The Investigator may choose to exclude patients who have had complications resulting from prior lumbar punctures.
. Patient history of poorly controlled seizure disorder.