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High Dose Ambroxol in Adult MPS III
NCT06614894Phase 3Recruiting
- Start
- Dec 2024
- Primary completion
- May 2026
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of Ambroxol hydrochloride oral solution in Sanfilippo syndrome. Primary endpoint: Safety and Tolerability at 52 weeks. Started Dec 2024; status: recruiting.
02
Drugs
Ambroxol hydrochloride oral solution
03
Conditions
Sanfilippo syndrome
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