NCT07285369Completed
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Ambroxol in Type III Gaucher Disease (GD3): A Prospective 6-Month Single-Center Open-Label Study With an Optional 12-month Extension Phase
Source: ClinicalTrials.gov · Last updated 24 Aug 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Interventional
- Enrollment
- 12 (actual)
- First submitted
- 21 Nov 2025
- Start date
- 1 Mar 2025
- Primary completion date
- 30 Mar 2026
- Lead sponsor
- Agyany Pharma LTD
- Diseases
- Gaucher disease
- NCT number
- NCT07285369
What you commit to
- Study type: Interventional
- Participants enrolled: 12
- From 1 Mar 2025 to the primary completion date, 30 Mar 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Recent chaperone therapy or investigational treatment within the last 6 months
Other illness
- Life-threatening visceral disease (related or unrelated to Gaucher Disease)
- Clinically significant cardiovascular, gastrointestinal, pulmonary, neurologic, endocrine, or psychiatric conditions
- History of cancer, drug or alcohol abuse, major organ transplant, or inability to adhere to study requirements
Lab values
- Renal insufficiency (eGFR \< 30 mL/min/1.73 m²)
Other
- Blood transfusion dependency
- Serious swallowing difficulties
- Pregnancy or lactation
Who the study is looking for
- Pediatric patients aged 3 to 18 years
- Genetically confirmed Type III Gaucher Disease (GD3)
- Treatment naïve or receiving enzyme replacement therapy (ERT)
- SARA score ≥ 8
- Sexually active females must agree to use contraception
- All participants must not be pregnant or breastfeeding
Age in the registry: 3 years to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| The Children's Hospital Coordinator of this site? Confirm its status | Lahore | Pakistan | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.