In short
Clinical trials are investigating Ambroxol Hydrochloride in several patient groups, including people with Parkinson’s disease, Dementia with Lewy Bodies, and Gaucher disease type 3. These studies are looking at whether treatment can improve symptoms or disease-related measures, and they also assess clinical outcomes in specific trial phases.
Key points
- Clinical trials of Ambroxol Hydrochloride are being done in Parkinson’s disease, Dementia with Lewy Bodies, and Gaucher disease type 3. The studies are mostly Phase 2 and Phase 3, which means they are testing the treatment in patient groups and measuring clinical results. In Parkinson’s disease, the trial focuses on people with a GBA1 mutation and measures motor symptoms. In Dementia with Lewy Bodies, the trial looks at thinking, behavior, and daily function. In Gaucher disease type 3, the study measures a cerebrospinal fluid marker called Lyso-GL1.
Trial overview
The clinical trials listed here are studying Ambroxol Hydrochloride in people with different neurological and genetic conditions. The studies are interventional, which means researchers give a treatment and then measure the results.
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.
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. It is authorised and plans to enroll 80 participants.
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. The main outcome is the change in the MDS-UPDRS part III motor subscale in the practically defined OFF medication state at 60 weeks.
The trial compares oral Ambroxol Hydrochloride with microcrystalline cellulose, which is the control treatment in this study. The focus is on motor symptoms, meaning movement problems such as stiffness, slowness, or tremor measured by the study scale.
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. Prodromal means the early stage, when signs of disease may just be starting. The study is authorised and plans to enroll 156 participants.
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. The main outcome is the mean score on the MMSE from screening to 18 months in the treatment group compared with the control group.
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. This makes the trial important for understanding whether the treatment may help people in the early stages of this condition.
Gaucher disease type 3 study
A Phase 3 n-of-1 series is studying children and adults with Gaucher disease type 3. The study is authorised and has a very small enrollment of 4 participants.
The intervention is oral Ambroxol Hydrochloride, compared with lactose monohydrate in the trial record. The main outcome is the change in cerebrospinal fluid (CSF) Lyso-GL1 in participants receiving Ambroxol Hydrochloride.
CSF is the fluid around the brain and spinal cord, and Lyso-GL1 is a marker being measured in this study. 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.
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. In Parkinson’s disease, the endpoint is a motor score at 60 weeks. In Dementia with Lewy Bodies, the endpoint is the MMSE score over 18 months. In Gaucher disease type 3, the endpoint is a change in CSF Lyso-GL1.
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.
- Motor outcome: used in Parkinson’s disease to measure movement symptoms.
- Cognitive outcome: used in Dementia with Lewy Bodies to measure thinking and memory.
- Biomarker outcome: used in Gaucher disease type 3 to measure a disease-related substance in CSF.
Key patient groups
The trials are targeted to different patient groups, so not everyone with these diseases would be in the same study. The Parkinson’s disease study focuses on people with a GBA1 mutation. The Dementia with Lewy Bodies study focuses on new and early patients with prodromal or mild disease. The Gaucher disease type 3 study includes children and adults.
These details matter because clinical trials often have strict entry rules to make sure the right people are studied for the right question.