Ambroxol in New and Early DLB, A Phase IIa Multicentre Randomized Controlled Double Blind Clinica… (NCT04588285) | Clinical Trial Compass
RecruitingPhase 2
Ambroxol in New and Early DLB, A Phase IIa Multicentre Randomized Controlled Double Blind Clinical Trial
Norway180 participantsStarted 2021-05-04
Plain-language summary
This is a confirmatory investigational medicinal product (IMP) study to investigate the effects on cognition, functional decline and on neuropsychiatric symptoms of the Glucocerebrosidase (GCase) enhancing chaperone ambroxol in participants diagnosed with prodromal and early dementia with Lewybodies (DLB).
Who can participate
Age range50 Years – 85 Years
SexALL
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Inclusion criteria
✓. Male or female.
✓. Age ≥ 50 and ≤ 85 years of age.
✓. Confirmed diagnosis of Dementia with Lewy Bodies (DLB) or Mild Cognitive Impairment in DLB (DLB-MCI).
✓. MMSE score\>=15
✓. Able and willing to provide informed consent prior to any study related assessments and procedures at screening visit 1.
✓. Capable of complying with all study procedures.
✓. Willing to provide blood samples for genetic analyses of APOE and GBA.
✓. 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 BID (day 15-21), 315 mg TID (day 22-28) and 420 mg TID (day 29-550)).
Exclusion criteria
✕. Current treatment with anticoagulants (e.g. warfarin) that might preclude safe completion in the opinion of the Investigator.
✕. Current use of investigational medicinal product or participation in another interventional clinical trial or who have done so within 30 days prior to the first dose in the current study.
✕. Exposure to more than three investigational medicinal products within 12 months prior to the first dose in the current study;
✕. Confirmed dysphagia that would preclude self-administration of ambroxol up to 6 tablets daily for the duration of day 1 to day 550/Month 18.
What they're measuring
1
Change in the incidence, nature and severity of AE's and SAE's from baseline.
Timeframe: All patient visits including phonecalls trough study completion, planned duration 18 months
2
Change in the number of participants with treatment discontinuations and study discontinuation due to AEs from baseline.
Timeframe: All patient visits including phonecalls trough study completion, planned duration 18 months
3
Change in the number of participants with electrocardiogram (ECG) abnormalities.
Timeframe: Through study completion at the following visits: Screening, Baseline, week 4, week 24, week 36, week 52, month 15, and month 18.
4
Change in blood analyses from baseline over time abnormalities.
Timeframe: Through study completion at the following visits: Screening, week 4, week 8, week 24, week 36, week 52, month 15, and month 18.
5
Change in MMSE-NR3 (Mini Mental Status Examination, Norwegian revised version) over time.
Timeframe: Through study completion at the following visits: Screening, week 24, week 36, week 52, Month 18.
6
ADCS-CGIC (Clinician's Global Impression of Change)
Timeframe: Through study completion at the following visits: Screening, week 24, week 36, week 52, month 18.
✕. Significant known lower spinal malformations or other spinal abnormalities that would preclude lumbar puncture.
✕. History of known sensitivity to the study medication, ambroxol or its excipients (lactose monohydrate, granulated microcrystalline cellulose, copovidone and magnesium stearate) in the opinion of the investigator that contraindicates their participation.
✕. History of known rare hereditary disorders of galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption.
✕. History of illegal substance abuse, drug abuse or alcoholism in the opinion of the Investigator that would preclude participation in the study.
Change in CDR-SB (Clinical Dementia Rating-Sum of Boxes).
Timeframe: Through study completion at the following visits: Screening, week 24, week 36 week 52, Month 18.
8
Change NPI (neuropsychiatric inventory)
Timeframe: Through study completion at the following visits: Screening, week 24, week 36, week 52, month 18.
9
GDS (geriatric depression scale) - 15 items
Timeframe: Through study completion at the following visits: Screening, week 24, week 36, week 52, month 18.