Clinical Trials Logo

Clinical Trial 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).


Clinical Trial Description

Participants will be recruited through established network of Norwegian Memory Clinics. Patients will be randomised to ambroxol with proven effect on the lysosomal and glucocerebrosidase pathology in DLB or placebo. The randomization will be stratified based on APOE e4 and on the concentration of A-beta in CSF. The frequency of GBA genotypes in the active treatment and placebo groups will be calculated at study end. The blinded phase will last for 18 months and an open extension with ambroxol will be offered to all participants for one additional year. The primary outcomes will be cognition, global function, disease stage, progression, and neuropsychiatric symptoms. Secondary outcomes will be on sleep disturbances, falls, fluctuations and parkinsonism, and exploratory outcomes will be impact on the potential biomarkers for drug effects defined as qEEG, DaTSCAN, MRI and α-synuclein in CSF. One hundred and eighty participants will be recruited in total. Each participant will orally self-administer or administer by a caregiver ambroxol or placebo at 5 intra-participant dose escalations 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)).Participants will be subjected to clinical and laboratory assessments to assess the safety, tolerability effects of ambroxol on blood biomarkers and MRI, DaTSCAN, ECG, EEG and lumbar puncture. Each participant will undergo 8 hospital visits and 16 telephone visits for the blinded phase of the study during the first 18 months. Hospital visits will additionally include 1 or 2 screening appointments within 60 days of Day 1 hospital visit (at which participants will receive the first dose of ambroxol), followed by visits at week 4, week 8, week 24, week 36, week 52, month 15 and month 18. Participants will receive a telephone call 3 days after lumbar puncture to record any complaints. Participants will receive 16 telephone calls to record any drug related adverse events in between hospital visits, between 1-3 days before and after each dose escalation (day 1, 8, 15, 22 and 29, week 12,16, 20, 28, 32, 40, 44, 48 and month 13, 14, 16 and 17). All participants will be offered treatment with the IMP for 12 additional months from month 18 - month 30. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04588285
Study type Interventional
Source Helse Fonna
Contact Arvid Rongve, Phd
Phone 90548749
Email arvid.rongve@helse-fonna.no
Status Recruiting
Phase Phase 2
Start date May 4, 2021
Completion date July 1, 2025

See also
  Status Clinical Trial Phase
Recruiting NCT03996460 - K0706 for Patients Diagnosed With Dementia With Lewy Bodies Phase 2
Recruiting NCT04335994 - ENhancing Outcomes in Cognitive Impairment Through Use of Home Sleep ApNea Testing N/A
Recruiting NCT02194816 - Modifiable Variables in Parkinsonism (MVP)
Active, not recruiting NCT01208675 - The Swedish BioFINDER Study
Completed NCT03538522 - A Double-Blind, Placebo-Controlled Safety and Efficacy Study of NA-831 Phase 2
Recruiting NCT03672448 - The China Longitudinal Aging Study of Cognitive Impairment
Completed NCT01340001 - Effects of Nucleus Basalis of Meynert Stimulation on Cognitive Disorders in Dementia With Lewy Bodies N/A
Not yet recruiting NCT04760860 - Terazosin for Dementia With Lewy Bodies Phase 1/Phase 2
Completed NCT02910102 - Study Evaluating Intepirdine (RVT-101) on Gait and Balance in Subjects With Dementia Phase 2
Terminated NCT02928445 - Long-Term Extension Study of Intepirdine (RVT-101) in Subjects With Dementia With Lewy Bodies: HEADWAY-DLB Extension Phase 2/Phase 3
Terminated NCT02871427 - Open-label Study of Nelotanserin in Lewy Body Dementia With Visual Hallucinations or REM Sleep Behavior Disorder Phase 2
Completed NCT05188105 - Alpha tACS in Dementia With Lewy Bodies N/A
Active, not recruiting NCT04167813 - Trial of Ondansetron as a Parkinson's HAllucinations Treatment Phase 2
Enrolling by invitation NCT04954183 - Development of an EEG Diagnostic for Alzheimer's Disease
Recruiting NCT05326750 - Non-invasive Neurostimulation as a Tool for Diagnostics and Management for Neurodegenerative Diseases N/A
Completed NCT01023672 - "Pilot Study of Armodafinil in Patients With Dementia With Lewy Bodies" Phase 4
Completed NCT03907748 - Home-based Family Caregiver-delivered Music and Reading Interventions for People With Dementia N/A
Completed NCT04649164 - Learning to PERSEVERE: Peer Mentor Support and Caregiver Education in Lewy Body Dementia N/A
Recruiting NCT03924414 - Trial of Parkinson's And Zoledronic Acid Phase 4
Completed NCT05885620 - SAMi Intervention Study to Evaluate Smartwatch Interventions in Persons With MCI and Dementia N/A