Ignite Creation Date:
2025-12-24 @ 11:03 PM
Ignite Modification Date:
2026-01-05 @ 5:41 PM
Study NCT ID:
NCT07285369
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-12-16
First Post:
2025-11-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
Sponsor:
Agyany Pharma LTD
Organization:
Study Overview
Official Title:
Ambroxol in Type III Gaucher Disease (GD3): A Prospective 6-Month Single-Center Open-Label Study With an Optional 12-month Extension Phase
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
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:
This single-center, prospective, open-label study investigates the safety, tolerability, and efficacy of high-dose Ambroxol in pediatric patients with genetically confirmed Type III Gaucher Disease (GD3). The study will enroll 12 participants aged 3 to 18 years, either treatment naïve or receiving enzyme replacement therapy (ERT). Participants will receive high-dose Ambroxol orally (mean 35 mg/kg bodyweight) over a 6-month period, with an optional 12-month extension.
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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: