An interventional study of Ambroxol in Gaucher Disease, Type 3, sponsored by Agyany Pharma LTD. Completed at 1 site in Pakistan. Open to participants aged 3 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-08-24.
Sponsored by Agyany Pharma LTD · Not applicable, Interventional, and Treatment
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.
This single-center, prospective, open-label study investigates the safety, tolerability, and efficacy of high-dose Ambroxol in pediatric patients with genetically confirmed Type III Gaucher Disease (GD3). The study will enroll 12 participants aged 3 to 18 years, either treatment naïve or receiving enzyme replacement therapy (ERT). Participants will receive high-dose Ambroxol orally (mean 35 mg/kg bodyweight) over a 6-month period, with an optional 12-month extension.
Primary Objective:
Evaluate the safety and tolerability of high-dose Ambroxol administered with or without ERT.
Secondary Objective:
Assess efficacy based on at least a 20% improvement in at least 50% of participants using the following measures:
Intervention:
High-dose Ambroxol administered orally (mean 35 mg/kg bodyweight)
Study Location:
The Children's Hospital, Lahore, Pakistan
This study aims to provide preliminary safety and efficacy data on Ambroxol as a therapeutic option for pediatric patients with GD3, potentially informing future larger-scale clinical trials.
Exclusion Criteria:
All participants will receive high-dose Ambroxol orally at a mean dose of 35 mg/kg bodyweight daily for 6 months, with an optional 12-month extension. The drug may be administered with or without enzyme replacement therapy (ERT).
Drug: 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).
Safety and Tolerability of High-Dose Ambroxol
Incidence and Severity of Treatment-Emergent Adverse Events
Time frame: 6 months (with optional assessment at 12-month extension)
Assess the efficacy of high-dose (mean 35mg/kg bodyweight) Ambroxol by at least 20% improvement in at least 50% of the patients measured with: assessment and Rating of Ataxia (SARA) scale for patients with ataxia.
50% of Participants Achieving ≥20% Improvement in SARA Score
Time frame: 6 months (with optional assessment at 12-month extension)
Plan to share: Undecided
No publications or documents are linked to this record.
This study is completed, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Agyany Pharma LTD