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.
177 studies on the registry are indexed under Gaucher Disease; 41 are open to participants now.
This study's enrollment of 12 is below the median of 20 across 100 interventional studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Agyany Pharma LTD is the lead sponsor of 2 studies on the registry; none are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Ran from Mar 2025 to primary completion in Mar 2026, 12 months.
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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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Studies of the drug tested here: Ambroxol
Agyany Pharma LTD