NCT06614894View on ClinicalTrials.gov

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

PHASE2/PHASE3RecruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

December 1, 2024

Primary Completion Date

May 1, 2026

Study Completion Date

June 30, 2026

Conditions
Sanfilippo SyndromeMPS3
Interventions
DRUG

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

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

DRUG

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

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

DRUG

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

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

Trial Locations (1)

22030

RECRUITING

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

All Listed Sponsors
collaborator

Team Sanfilippo

UNKNOWN

lead

Ozlem Goker-Alpan

OTHER