Table of Contents

• Trial overview
• Parkinson’s disease study
• Dementia with Lewy Bodies study
• Gaucher disease type 3 study
• Trial designs and endpoints
• Key patient groups

Trial overview

The clinical trials listed here are studying Ambroxol Hydrochloride in people with different neurological and genetic conditions.^[1][2][3] The studies are interventional, which means researchers give a treatment and then measure the results.^[1][2][3]

Most of the trials are in Phase 2 or Phase 3, so they are looking at how the treatment performs in real patient groups and what clinical changes may happen over time.^[1][2][3]

Parkinson’s disease study

The GREAT trial, also called the GRoningen Early-PD Ambroxol Treatment Trial, is a Phase 2 study in people with Parkinson’s disease.^[1] It is authorised and plans to enroll 80 participants.^[1]

This study is designed to look at the disease-modifying properties of Ambroxol Hydrochloride in people with a GBA1 mutation, which is a change in a gene linked to Parkinson’s disease risk in this trial description.^[1] The main outcome is the change in the MDS-UPDRS part III motor subscale in the practically defined OFF medication state at 60 weeks.^[1]

The trial compares oral Ambroxol Hydrochloride with microcrystalline cellulose, which is the control treatment in this study.^[1] The focus is on motor symptoms, meaning movement problems such as stiffness, slowness, or tremor measured by the study scale.^[1]

Dementia with Lewy Bodies study

This Phase 2 multicentre randomized controlled double blind trial is studying new and early patients with prodromal and mild Dementia with Lewy Bodies.^[2] Prodromal means the early stage, when signs of disease may just be starting.^[2] The study is authorised and plans to enroll 156 participants.^[2]

The trial compares oral Ambroxol Hydrochloride with a placebo, which is a treatment that looks the same but does not contain the active study drug.^[2] The main outcome is the mean score on the MMSE from screening to 18 months in the treatment group compared with the control group.^[2]

The study is also looking at cognitive, neuropsychiatric, and functional outcomes, which means thinking skills, behavior and mental symptoms, and the ability to do daily activities.^[2] This makes the trial important for understanding whether the treatment may help people in the early stages of this condition.^[2]

Gaucher disease type 3 study

A Phase 3 n-of-1 series is studying children and adults with Gaucher disease type 3.^[3] The study is authorised and has a very small enrollment of 4 participants.^[3]

The intervention is oral Ambroxol Hydrochloride, compared with lactose monohydrate in the trial record.^[3] The main outcome is the change in cerebrospinal fluid (CSF) Lyso-GL1 in participants receiving Ambroxol Hydrochloride.^[3]

CSF is the fluid around the brain and spinal cord, and Lyso-GL1 is a marker being measured in this study.^[3] Because the trial is an n-of-1 series, it is designed around very small, individual-level treatment observations rather than a large group comparison.^[3]

Trial designs and endpoints

The trial designs are different, but each one is focused on a clear clinical endpoint, or main result to be measured.^[1][2][3] In Parkinson’s disease, the endpoint is a motor score at 60 weeks.^[1] In Dementia with Lewy Bodies, the endpoint is the MMSE score over 18 months.^[2] In Gaucher disease type 3, the endpoint is a change in CSF Lyso-GL1.^[3]

These endpoints show that the trials are not just checking whether treatment is given, but whether it changes a measured sign of disease or function.^[1][2][3]

• Motor outcome: used in Parkinson’s disease to measure movement symptoms.^[1]
• Cognitive outcome: used in Dementia with Lewy Bodies to measure thinking and memory.^[2]
• Biomarker outcome: used in Gaucher disease type 3 to measure a disease-related substance in CSF.^[3]

Key patient groups

The trials are targeted to different patient groups, so not everyone with these diseases would be in the same study.^[1][2][3] The Parkinson’s disease study focuses on people with a GBA1 mutation.^[1] The Dementia with Lewy Bodies study focuses on new and early patients with prodromal or mild disease.^[2] The Gaucher disease type 3 study includes children and adults.^[3]

These details matter because clinical trials often have strict entry rules to make sure the right people are studied for the right question.^[1][2][3]