High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2027-01-01
Target enrollment:
Participant gender:
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 nave 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.