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Study identifier: NCT06614894 Synced from ClinicalTrials.gov · July 29, 2026
● 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

Condition: Sanfilippo Syndrome · MPS3  ·  Sponsor: Ozlem Goker-Alpan

PhasePhase 2/Phase 3
Planned participants10
Who can joinAll sexes, 18 Years to 99 Years
Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Arooj Agha  ·  571-732-4575  ·  aagha@ldrtc.org

Lauren Noll  ·  571-732-4655  ·  lnoll@ldrtc.org

Always discuss trial participation with your own doctor first.

Locations (1)

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

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Source record: clinicaltrials.gov/study/NCT06614894