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 range3 Years – 18 Years

SexALL

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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

What they're measuring

Safety and Tolerability of High-Dose Ambroxol

Timeframe: 6 months (with optional assessment at 12-month extension)

Trial details

NCT IDNCT07285369

SponsorAgyany Pharma LTD

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-03-30

View on ClinicalTrials.gov More Gaucher Disease, Type 3 trials