GRoningen Early-PD Ambroxol Treatment

Launched by UNIVERSITY MEDICAL CENTER GRONINGEN · Apr 13, 2023

Keywords

ClinConnect Summary

The GRoningen Early-PD Ambroxol Treatment trial is studying whether a medication called ambroxol can help improve symptoms or slow down the progression of Parkinson's Disease (PD) in patients with a specific genetic mutation known as GBA1. This trial is important because the GBA1 mutation affects a protein that plays a role in the disease. By giving participants ambroxol, researchers hope to see if it can positively impact the way the disease develops. The trial will involve patients undergoing various tests, including motor skills, thinking abilities, and scans, to gather information about their health.

To participate in this trial, you need to have a diagnosis of Parkinson's Disease for 10 years or less and carry the GBA1 mutation. It's important that you are able to understand the study and consent to participate, as well as take the medication or a placebo (an inactive pill) by yourself. If you're interested in joining, you should also be aware that certain conditions, like having severe swallowing difficulties or specific health issues, might prevent you from being eligible. The trial is currently recruiting participants aged 65 to 74, and both men and women can join.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosis of Parkinson's disease, according to Movement Disorders Society (MDS) criteria (27)
• • Disease duration of 10 years or less at time of inclusion
• • PD patients carrying a GBA1 mutation
• • Able to write written informed consent, understanding study protocol and perform protocol related actions
• • Willing and able to self-administer oral ambroxol or placebo medication
• Exclusion Criteria:
• • The refusal to be informed about an unforeseen clinical finding
• • Use of an implanted Deep Brain Stimulation (DBS) system
• • Confirmed dysphagia that would preclude self-administration of ambroxol or placebo tablets
• • History of known sensitivity to the study medication
• • Pregnant or breastfeeding women
• • Participants of childbearing potential that would not use adequate birth control, consisting of a negative pregnancy test at the screening visit and use of accepted contraceptive methods defined as highly effective while participating in the study
• • MRI incompatible implants in the body
• * Any clinically significant or unstable medical or surgical condition that in the opinion of the principal investigator may put the participant at risk when participating in the study or may influence the results of the study or affect the participant's ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG), or laboratory tests. Such conditions may include:
• • 1. Impaired renal function (a positive urine dipstick test, and laboratory values below or above: a eGFR \<45 ml/min 1,73M2, Sodium 135-145 mmol/L, Potassium 3.5-5.0 mmol/L, Urea 2.5-7.5mmol/L).
• • 2. Moderate/severe hepatic impairment (laboratory values below or above: ASAT 0- 80U/L, ALAT0-90 U/L, GGT \> 80 U/L, Alkaline Phosphatase 35-210 U/L).