Status: Recruiting
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
A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
Participants needed: 10
Trial details
Phase: Phase 2, Phase 3Age: 18-99Biological sex: AllType: InterventionalSponsor: Ozlem Goker-AlpanUpdated: Aug 20, 2025Locations: 1
Eligibility criteria
IRB - approved informed consent/assent signed by subject and/or parent(s) or leg... [+6]
Unwilling or unable to follow protocol requirements as per principal investigato... [+10]