High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)

Summary

Type: Prospective, open-label, single center study Duration: 6 months with an optional 12-month extension phase Participants: 12 pediatric patients diagnosed with type III Gaucher disease (GD3) aged ≥3 to ≤18 years old treatment naïve or on enzyme replacement therapy (ERT). They will be treated with high-dose Ambroxol (mean 35mg/kg bodyweight). Location: The Children's Hospital, Lahore, Pakistan.

Timeline

Start

2025-03-01

Primary completion

2026-03-30

Completion

2026-03-30

Drugs

Indications