High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) (NCT07285369) | Clinical Trial Compass
TerminatedNot Applicable
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Stopped: Sponsor stopped the study
Pakistan12 participantsStarted 2025-03-01
Plain-language 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.
Who can participate
Age range
3 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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
Exclusion Criteria:
* Life-threatening visceral disease (related or unrelated to Gaucher Disease)
* Blood transfusion dependency
* Clinically significant cardiovascular, gastrointestinal, pulmonary, neurologic, endocrine, or psychiatric conditions
* Serious swallowing difficulties
* Renal insufficiency (eGFR \< 30 mL/min/1.73 m²)
* Recent chaperone therapy or investigational treatment within the last 6 months
* Pregnancy or lactation
* History of cancer, drug or alcohol abuse, major organ transplant, or inability to adhere to study requirements
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety and Tolerability of High-Dose Ambroxol
Timeframe: 6 months (with optional assessment at 12-month extension)