RecruitingPhase 2Phase 3NCT06614894

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


Sponsor

Ozlem Goker-Alpan

Enrollment

10 participants

Start Date

Dec 1, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

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


Eligibility

Min Age: 18 YearsMax Age: 99 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, a drug called Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day, and others for people with mps3 and sanfilippo syndrome. The study is currently recruiting participants at 1 location. People eligible for this study include aged 18 Years to 99 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGAmbroxol Hydrochloride 30 mg tablet - 9 mg/kg/day

Ambroxol Hydrochloride 30 mg oral pill/tablet - 9 mg/kg/day

DRUGAmbroxol Hydrochloride 30 mg tablet - 18 mg/kg/day

Ambroxol Hydrochloride 30 mg oral pill/tablet - 18 mg/kg/day

DRUGAmbroxol Hydrochloride 30 mg tablet - 27 mg/kg/day

Ambroxol Hydrochloride 30 mg oral pill/tablet - 27 mg/kg/day


Locations(1)

Lysosomal & Rare Disorders Research & Treatment Center, Inc.

Fairfax, Virginia, United States

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NCT06614894


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