The Dutch Parkinson GBA Ambroxol trial

Phase 1

• Conditions: Parkinson's disease with a GBA1 mutation; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2021-001833-38-NL
• Lead Sponsor: niversity Medical Center Groningen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-03-31
• Last Update Posted: 26 February 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

In order to be eligible to participate in this study, a subject must meet all of the following criteria:
-Diagnosis of Parkinson’s disease, according to Movement Disorders Society (MDS) criteria <10 years
-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 medication

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion Criteria

A potential subject who meets any of the following criteria will be excluded from participation in this study:
-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 child bearing potential that would not use adequate birth control 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
2. Moderate/severe hepatic impairment
3. A major cardiovascular event (e.g. myocardial infarction, acute coronary syndrome, decompensated congestive heart failure, pulmonary embolism, coronary revascularisation that occurred within 6 months prior to the screening visit.)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified