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

Recruiting

Phase 2 — Testing in a larger group (up to a few hundred people) to further evaluate safety and begin to assess effectiveness.

Trial locations(1 site)

United States

Lysosomal & Rare Disorders Research & Treatment Center, Inc., Fairfax, Virginia