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)

🧪 Interventions in This Study

• Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day (DRUG)
• Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day (DRUG)
• Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day (DRUG)

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (1 Sites)

• Lysosomal & Rare Disorders Research & Treatment Center, Inc. — Fairfax, Virginia

📞 Contact the Study Coordinator

Phone: (571) 732-4575

Email: aagha@ldrtc.org

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

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💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Contact the study coordinator using the phone/email above.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.