LiO-AD: Lithium Orotate in Alzheimers Disease Feasibility, Biomarker Engagement, and Clinical Res… (NCT07459959) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
LiO-AD: Lithium Orotate in Alzheimers Disease Feasibility, Biomarker Engagement, and Clinical Response
40 participantsStarted 2026-10-01
Plain-language summary
The goal of this clinical trial is to assess feasibility, safety, tolerability, and central nervous system target engagement of oral lithium orotate in adults with biomarker-confirmed early Alzheimer's disease. The main questions it aims to answer are:
* Can participants be recruited, retained, and remain adherent (target ≥80%) over 9 weeks of treatment, and what is the frequency and severity of adverse events?
* Does lithium orotate increase cerebrospinal fluid (CSF) lithium concentration from baseline to 9 weeks compared with placebo? Researchers will compare daily lithium orotate to matched placebo to see if lithium orotate demonstrates acceptable feasibility, safety, and tolerability and engages the central nervous system target (CSF lithium).
Participants will:
* Be randomized in a double-blind manner to receive lithium orotate or placebo for 9 weeks, with titration from week 1: 240 mg/day (10mg elemental lithium) to week 2: 480 mg/day (20mg elemental lithium) and week 3: 720 mg/day (30mg elemental lithium) if tolerated; dose reductions are permitted for side effects.
* Attend study visits for safety monitoring, adherence support (caregiver pill logs/diaries), and review of concomitant medications and adverse events.
* Provide blood samples and undergo lumbar punctures at baseline and post-treatment to measure CSF and serum lithium and Alzheimer's-related biomarkers; complete brief cognitive testing and neuropsychiatric symptom assessments.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Diagnosis of Alzheimer's disease confirmed by biomarkers (imaging or biofluid evidence of amyloid-beta and tau pathology)
* Mild stage of Alzheimer's disease: Clinical Dementia Rating (CDR) ≤ 1 or Quick Dementia Rating System (QDRS) ≤ 8
* Medically stable and able to attend study visits and complete study procedures
* On stable doses of any psychotropic medications for at least 4 weeks before the baseline visit
* Not currently receiving anti-amyloid monoclonal antibody therapy
Exclusion Criteria:
* New or unstable neurological disorder or unstable psychiatric illness that could affect safety or study results
* Clinically significant kidney or thyroid problems that pose safety concerns, or abnormal safety labs judged to be related to study drug and requiring discontinuation
* Use of thiazide diuretics during the dosing period (unless stopped at least 4 weeks before baseline)
* Chronic daily use of non-aspirin NSAIDs (including COX-2 inhibitors); short courses require study team approval and may require temporary study drug hold and safety labs before resuming
* Starting excluded therapies during the active treatment period (e.g., anti-amyloid monoclonal antibody treatment)
* Noncompliance with essential study procedures that would prevent collection of primary safety or feasibility endpoints (e.g., repeated missed visits or refusal of critical labs)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is still in Phase 1/2 and hasn't started recruiting yet — what does that mean for how much is already known about whether lithium orotate is safe and effective for Alzheimer's disease, and how does that uncertainty weigh against other options available to me right now?
2The trial is specifically monitoring kidney and thyroid function as safety outcomes — given my current health history, are there any existing issues with my kidneys or thyroid that would make participating in this study riskier for me than for someone else?
3Since this study is primarily designed to test whether the trial itself is feasible — things like whether people stay enrolled and take the medication as directed — rather than to prove the treatment works, is it reasonable to expect any personal medical benefit from joining, or would I mainly be contributing to research for future patients?
4The trial isn't recruiting yet, and given that it's a Phase 1/2 study that will still be figuring out dosing and tolerability, how long might it realistically be before it opens and then completes, and does waiting for it make sense compared to starting an approved treatment now?
5Because the study is closely tracking dose modifications and dropouts as key measures, what kind of time commitment and follow-up visits would likely be involved, and is that realistic given my current caregiving situation or ability to travel to the study site?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Feasibility (Recruitment, Retention)
Timeframe: Baseline up to Week 9
2
Feasibility (Visit Completion)
Timeframe: Baseline up to Week 9
3
Feasibility (Adherence)
Timeframe: Baseline up to Week 9
4
Safety (Frequency of Adverse Events)
Timeframe: Baseline up to Week 11 (includes Safety Follow-up)
5
Safety (Adverse Events Relatedness)
Timeframe: Baseline through Week 11 (includes Safety Follow-up)
6
Safety (Adverse Events Severity)
Timeframe: Baseline through Week 11 (includes Safety Follow-up)
7
Safety (Renal function)
Timeframe: Baseline through Week 11 (includes Safety Follow-up)