Table 2.
Inclusion criteria | 1. Age ≥ 50 and ≤ 85 years of age, both gender |
2. Confirmed diagnosis of DLB including mild cognitive impairment in DLB (DLB-MCI). | |
3. MMSE score >=15 at screening | |
4. Able and willing to provide informed consent prior to any study related assessments and procedures | |
5. Capable of complying with all study procedures | |
6. Willing to provide blood samples for genetic analyses of APOE and GBA | |
7. Willing and able to self-administer or administer by a caregiver oral ambroxol medication, from day 1 to study end (at 60 mg TID (day 1-7), 120 mg TID (day 8- 14), 315 mg BID (day 15-21), 315 mg TID (day 22-28) and 420 mg TID (day 29-550)). | |
8. Contact with caregiver at least 3 times a week, to ensure sufficient information from the caregiver regarding participant’s status and possible change in condition | |
9. Able to travel to the participating study site | |
10. A female participant is eligible to participate if she is of non-childbearing potential or if women is of childbearing potential must use accepted contraceptive methods | |
11. Caregiver needs to be ≥ 18 years when signing the informed consent form | |
Exclusion criteria | 1. Current treatment with anticoagulants (e.g. warfarin, argatroban, dabigatraneteksilat, rivaroksaban, apiksaban, edoksaban) |
2. Current use of IMP or participation in another interventional clinical trial | |
3. Exposure to more than three IMP within 12 months prior to the first dose in the current study | |
4. Confirmed dysphagia that would preclude self-administration of ambroxol up to six tablets/d for the duration of this study | |
5. Known sensitivity to the study medication, ambroxol or its excipients (lactose monohydrate, granulated microcrystalline cellulose, silicon dioxide, magnesium stearate and denatonium benzoate) | |
6. History of known rare hereditary disorders of galactose intolerance: lactase deficiency or glucose-galactose malabsorption | |
7. History of severe substance abuse (drug abuse or alcohol) | |
8. Donation of blood (1 unit or 350 ml) within three months prior to receiving the first dose of the study drug | |
9. Pregnant or breastfeeding | |
10. Any clinically significant or unstable medical or surgical condition that may put the participant at risk when participating or may influence the results of the study or affect the participant’s ability to take part in the study. Such conditions may include: | |
a) impaired renal function defined by eGFR<=30 | |
b) moderate/severe hepatic impairment defined by Child-Pugh score >1 | |
c) 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) | |
d) Major stroke | |
e) Major depression defined clinically or by GDS-15 >=11 points or delirium or psychotic disorder unrelated to DLB. | |
f) Cancer, history of metastatic cancer, terminal illness or clinically significant disease within ≤5 years, except for adequately treated basal cell skin cancer. | |
11. Planned major surgical treatment during the study period |
APOE, apolipoprotein epsilon; BID, twice a day; DLB, dementia with Lewy bodies; DLB-MCI, mild cognitive impairment in DLB; eGFR, estimated glomerular filtration rate; GDS, the Geriatric Depression Scale; IMP – investigational medicinal product, PD, Parkinson’s disease, PDD, dementia in PD; MMSE, Mini Mental State Examination; TID, three times a day.