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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

NCT06614894 · Status: RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2025-08-20

No results posted yet for this study

Summary

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

Conditions

  • Sanfilippo Syndrome
  • MPS3

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

Sponsors & Collaborators

  • Team Sanfilippo

    collaborator UNKNOWN

  • Ozlem Goker-Alpan

    lead OTHER

Principal Investigators

  • Ozlem Goker-Alpan, MD · Lysosomal & Rare Disorders Research & Treatment Center, Inc.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-01
Primary Completion
2026-05-01
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06614894 on ClinicalTrials.gov