Repurposing Nucleoside Reverse Transcriptase Inhibitors for Treatment of AD

Recruiting now · Phase 1 · Has a placebo group

Conditions studied: Alzheimer Disease, Early Onset, Mild Cognitive Impairment, Moderate Dementia

In brief

This is a randomized, double-blind clinical trial of a daily oral dose of 200 mg emtricitabine vs. placebo in 35 participants with biomarker-confirmed MCI or mild to moderate dementia due to Alzheimer's disease. Study duration for each subject participating in the placebo-controlled research study will be approximately 12 months (up to a 3 months Screening Period, Baseline visit (1 month), 6 months of placebo or emtricitabine dosing, and 1 month follow-up). Participants will have up to 2 months to complete all procedures for the month 6 study visit.

Key facts

Study ID: NCT04500847
Run by: Butler Hospital
People needed: 35
Starts: 2021-12-17
Expected to finish: 2026-03-31
Last updated by the study team: 2025-05-04

Who can join

Age: 50 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female, ages 50-85 years inclusive
Intellectually, visually and auditory capable, fluent in, and able to read, the language in which study assessments are administered (e.g. completion of at least six years of regular schooling or sustained employment or equivalent local level of knowledge).
Must meet NIA-AA research criteria for MCI and mild dementia due to AD
Mini Mental State Exam (MMSE) 15-30 inclusive
Clinical Dementia Rating (CDR) 0.5 - 2
Must meet a cerebrospinal fluid (CSF) pTau/Aβ42 ratio of > 0.024
Participants must have an appropriate study partner who agrees to participate in the study and who is intellectually, visually, and auditory capable, and fluent in, and able to read, the language in which study assessments are administered. Additionally, the study partner must be capable of and willing to: Accompany the participant to visits that requires the input of the study partner
Concurrent treatment with cholinesterase inhibitors and memantine are permitted on a stable dose for at least 60 days prior to baseline.

You may not qualify if…

Current medical or neurological condition that might impact cognition or performance on cognitive assessments, e.g., Huntington's disease, Parkinson's disease, syphilis, schizophrenia, bipolar disorder, active major depression, attention deficit/ hyperactivity disorder (ADD/ADHD), multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), active seizure disorder, current alcohol/drug abuse or dependence, or dependence within the last two years, or history of traumatic brain injury associated with loss of consciousness and ongoing residual transient or permanent neurological signs/symptoms including cognitive deficits, and/or associated with skull fracture
Brain MRI results showing findings unrelated to AD that, in the opinion of the investigator might be a leading cause of future cognitive decline, might pose a risk to the participant, or might confound MRI assessment for safety monitoring
Score "yes" on item four or item five of the Suicidal Ideation section of the Columbia Suicide Severity Rating Scale (eC-SSRS patient-reported outcome), if this ideation occurred in the past six months, or "yes" on any item of the Suicidal Behavior section, except for the "Non-Suicidal Self-Injurious Behavior" (item is included in the Suicidal Behavior section), if this behavior occurred in the past 2 years prior to screening
Use of other investigational drugs prior to screening until:
Small molecules: after five half-lives, or within 30 days until the expected pharmacodynamic effect has returned to baseline, whichever is longer
Biologicals: blood concentration has returned to baseline (or below serological responder threshold) for antibodies induced by active immunotherapy; or five half- lives for monoclonal antibodies or other biologicals
Approximately four weeks prior to randomization, the use of any drug or treatment known for the potential to cause major organ system toxicity, i.e. drugs that may require periodic safety monitoring of a specific organ or body fluid. Examples include, but are not limited to clozapine, cancer medical treatment like tamoxifen, systemic immunosuppressive drugs like methotrexate or interferon, or other immunosuppressive biological medicines for rheumatic diseases or multiple sclerosis
A positive drug screen, if, in the investigator's opinion, this is due to drug abuse or dependence.
Significant ECG findings that are assessed as clinically significant by the investigator (e.g. sustained ventricular tachycardia, significant second or third degree atrioventricular block without a pacemaker, long QT syndrome or clinically meaningful prolonged QT interval).
Contraindication to lumbar puncture including use of anti-coagulants, low platelet count, history of back surgery (with the exception of microdiscectomy or laminectomy over one level), signs or symptoms of intracranial pressure, spinal deformities or other spinal conditions that in the judgment of the investigator would preclude a lumbar puncture
History of or active hepatitis or HIV infection (based on a positive lab result for HBV and/or HIV, to be performed during screening
Severe renal impairment
Severe hepatic impairment
Significant cardiac disease including recent (within six months) myocardial infarction, congestive heart failure or unstable angina
Female subjects who are pregnant or currently breastfeeding.

Where it is running

Cedars-Sinai Medical Center — Los Angeles, California, United States (enrolling)
Memory and Aging Program, Butler Hospital — Providence, Rhode Island, United States (enrolling)

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.