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

Phase 2/3 Recruiting 10 enrolled Ozlem Goker-Alpan Team Sanfilippo · collab

Non-randomizedSequentialOpen-labelTreatment

Summary

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

Timeline

Start: 2024-12-01
Primary completion: 2026-05-01
Completion: 2026-06-30

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ambroxol	Small molecule	27 mg/kg	Oral

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

Summary

Timeline

Drugs

Indications