Ambroxol Hydrochloride for Sanfilippo Syndrome (MPS III)
This study is testing Ambroxol Hydrochloride in adults (18 years and older) with Sanfilippo Syndrome (MPS III). Researchers want to see how safe Ambroxol Hydrochloride is and how well your body handles different doses (9 mg/kg/day, 18 mg/kg/day, or 27 mg/kg/day). The main goal is to check for any side effects and how well the drug is tolerated over 52 weeks. You can join if you have a confirmed genetic diagnosis of MPS III. This is an open-label study, meaning both you and the study team will know which dose you are receiving. Ten participants are planned for this study.
- Study design
- This is an open-label dose escalation study, meaning you and the study team will know which dose of Ambroxol Hydrochloride you are receiving. It plans to enroll 10 adult participants.
- What's involved
- You would participate in a screening period, followed by 52 weeks of treatment with Ambroxol Hydrochloride, taken orally. This involves blood and urine collections, questionnaires, motor skills assessments, and evaluations by the study doctor.
- Compensation
- Not stated in the trial record.
- Follow-up
- After the 52-week treatment period, there will be a 4-week withdrawal and safety follow-up period.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Ozlem Goker-Alpan, MD · PRINCIPAL_INVESTIGATOR · Lysosomal & Rare Disorders Research & Treatment Center, Inc.
Who to contact
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What this trial measures
- Safety and TolerabilityFrom baseline to 52 weeks
1\. Safety and tolerability as measured by number of Participants with at least one serious and at least one non-serious Treatment Emergent Adverse Events (TEAEs), assessed by CTCAE v4.0.