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High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)

NCT07285369 · Status: TERMINATED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-04-03

No results posted yet for this study

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.

Conditions

  • Gaucher Disease, Type 3

Interventions

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).

Sponsors & Collaborators

  • Agyany Pharma LTD

    lead INDUSTRY

Principal Investigators

  • Huma Arshad Cheema, Prof. · The Children's Hospital, Lahore, Pakistan

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-03-01
Primary Completion
2026-03-30
Completion
2026-03-30

Countries

  • Pakistan

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07285369 on ClinicalTrials.gov