High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
NCT ID: NCT07285369
Last Updated: 2025-12-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
NA
12 participants
INTERVENTIONAL
2025-03-01
2027-01-01
Brief Summary
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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.
Detailed Description
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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:
* Assessment and Rating of Ataxia (SARA) for patients with ataxia
* Unified Myoclonus Rating Scale (UMRS) for patients with myoclonic epilepsy
* Lyso-Gb1 levels in peripheral blood after at least 6 months of treatment
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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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High-Dose Ambroxol
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).
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).
Interventions
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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).
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
3 Years
18 Years
ALL
No
Sponsors
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Agyany Pharma LTD
INDUSTRY
Responsible Party
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Principal Investigators
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Huma Arshad Cheema, Prof.
Role: PRINCIPAL_INVESTIGATOR
The Children's Hospital, Lahore, Pakistan
Locations
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The Children's Hospital
Lahore, , Pakistan
Countries
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Other Identifiers
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AGP3
Identifier Type: -
Identifier Source: org_study_id