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

• Status: RECRUITING
• Trial end date: 2026-06-30

Summary

A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).

• Phase: PHASE2

Details

• Lead Sponsor: Ozlem Goker-Alpan
• Collaborator: Team Sanfilippo
• Treatments: Ambroxol; Tablets