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High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Sponsor: Agyany Pharma LTD
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.
Official title: Ambroxol in Type III Gaucher Disease (GD3): A Prospective 6-Month Single-Center Open-Label Study With an Optional 12-month Extension Phase
Key Details
Gender
All
Age Range
3 Years - 18 Years
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2025-03-01
Completion Date
2026-03-30
Last Updated
2026-08-24
Healthy Volunteers
No
Conditions
Interventions
Ambroxol
High-dose Ambroxol will be administered orally at a mean dose of 35 mg/kg bodyweight daily. Participants will receive treatment for 6 months, with an optional 12-month extension. The drug may be given with or without concurrent enzyme replacement therapy (ERT).
Locations (1)
The Children's Hospital
Lahore, Pakistan