Clinical Trial

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

Terminated
View on ClinicalTrials.gov →
Why the trial stopped
Sponsor stopped the study
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.
Protocol Amendment History 1 amendment
This ClinicalTrials.gov record has been amended once since 2025-12-02.
Status change: Active, Not Recruiting → Terminated 2026-03-30
Trial Details
NCT Number NCT07285369
Lead Sponsor Agyany Pharma LTD
Conditions Gaucher Disease, Type 3
Enrollment 12 participants
Start Date 2025-03-01
Primary Completion 2026-03-30 (estimated)
Study Completion 2026-03-30 (estimated)
Updated on ClinicalTrials.gov 2026-04-03