A Phase 2/3 interventional study of Ambroxol Hydrochloride and Placebo in Parkinson and Parkinson Disease, sponsored by University Medical Center Groningen. Recruiting at 1 site in Netherlands. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-05-10.
Sponsored by University Medical Center Groningen · Phase 2/3, Interventional, and Treatment
The most common genetic risk factor for Parkinson's Disease is a heterozygous mutation of the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase). Reduced GCase activity is associated with aggregation of the protein alpha synucleine (aSyn) in the central nervous system, which is related to the pathological cause of PD. Ambroxol is a mucolytic expectorant that appears to facilitate the refolding of the misfolded GBA protein thats acts as a chaperone for GCase.
This randomized placebo-controlled trial aims to investigate the disease-modifying properties of ambroxol in PD patients with a GBA1-mutation. Patients will undergo motor and cognitive tests, as well as imaging and blood tests.
4,484 studies on the registry are indexed under Parkinson Disease; 1,081 are open to participants now.
This study's planned enrollment of 80 is above the median of 40 across 3,293 interventional studies indexed under Parkinson Disease.
Browse Parkinson Disease studies →University Medical Center Groningen is the lead sponsor of 609 studies on the registry; 174 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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:
Ambroxol 1800mg/day
Drug: Ambroxol Hydrochloride
Placebo
Drug: Placebo
Patients will either receive ambroxol or placebo. ambroxol will be given intially in a dosage of 600mg/day. After 1 week, this will be increased to 1200mg/day. After 2 weeks the maximum dosage of 1800mg/day will be given. In total, ambroxol will be administered for 48 weeks. This is followed by a 12 week washout period, after wich the final outcomes will be measured (week 60).
Patients will either receive ambroxol or placebo. ambroxol will be given intially in a dosage of 600mg/day. After 1 week, this will be increased to 1200mg/day. After 2 weeks the maximum dosage of 1800mg/day will be given. In total, ambroxol will be administered for 48 weeks. This is followed by a 12 week washout period, after wich the final outcomes will be measured (week 60).
MDS-UPDRS3 motor scale
Motor scale developed for PD patients, 0-132. 0 means good performance, 132 means very bad performance
Time frame: 60 weeks
Safety and tolerability measured by incidence of adverse events and possible side effects
AE will be monitored and patients will be questioned about side effects every week during the first 3 weeks and after that, every 3 months during the visits
Time frame: all throughout the study. specifically at: 1, 2, 3, 12, 24, 36, 48, 60 weeks
Glucocerebrosidase (GCase) activity in blood mononuclear cells
Measured by the level of sphingolipids in PBMCs
Time frame: 0, 12, 60 weeks
Striatal F-DOPA uptake as measured by [18] F-DOPA PET scan
Time frame: 0, 60 weeks
fMRI resting state to investigate the functional architecture and structural MRI for PET-scan
Fluctuations in the BOLD signal can be used to investigate the functional architecture and connectivity within the brain.
Time frame: 0, 60 weeks
Quality of Life (PDQ-39 questionnaire)
Time frame: 0, 60 weeks
Non Motor Symptoms (NMSS scale)
minimum value is 0, maximum value is 360. 0 indicating a good performance, 360 indicating a very bad performance
Time frame: 0, 60 weeks
Cognition, using the Montreal Cognitive Assessment (MoCA)
Range is 0-30, 0 indicating the worst performance, 30 indicating the best performance
Time frame: 0, 60 weeks
Plan to share: No
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University Medical Center Groningen