Phase 2
N=12
Pilot Study to Investigate the Safety and Feasibility of AntiRetroviral Therapy for Alzheimer's Disease
Alzheimer Disease, Early Onset
Bottom Line
View on ClinicalTrials.gov: NCT04552795 ↗Enrolled (actual)
12
Serious AEs
8.3%
Results posted
Aug 2024
Primary outcome: Primary: Change in Reverse Transcriptase Activity From Baseline to 24 Weeks in Plasma of Study Participants — -0.0074 Enzyme units
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- 3TC (Drug)
- Age
- Adult, Older Adult · 50+ yrs
- Sex
- All
- Sponsor
- The University of Texas Health Science Center at San Antonio
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Reverse Transcriptase Activity From Baseline to 24 Weeks in Plasma of Study Participants |
-0.0074 | — |
| PRIMARY 3TC CNS Penetration |
0.502 | — |
| SECONDARY Change in Dementia Severity From Baseline to Week 24 of Treatment Based on the PACC-5 Z-score |
-1.59; -1.59 | — |
| SECONDARY Incidence of Treatment-Emergent Adverse Events |
1; 11 | — |
| SECONDARY Incidence of Treatment-Emergent Abnormal Vital Signs |
— | — |
Summary
The objective of the study is to evaluate the ability of (-)-L-2',3'-dideoxy-3'-thiacytidine (3TC) to engage its intended target, penetrate the central nervous system (CNS), suppress neurodegeneration, and assess safety and tolerability in patients with early stage Alzheimer's disease. This study will provide the initial data on target engagement and Alzheimer's disease-relevant outcomes for future trials.
Eligibility Criteria
Inclusion Criteria
- Aged 50-99 years
- Clinical diagnosis of early Alzheimer's disease (Clinical Dementia Rating (CDR) = 0.5, Mini-Mental State Exam (MMSE) = 24-30)
- If using drugs to treat symptoms related to Alzheimer's disease, doses must be stable for at least eight weeks prior to screening visit 1
- Labs: Adequate blood cell counts (white blood cells: 4,000-111,000 cells per microliter (cells/mcL); absolute neutrophil count: 1,800-8,700 cells/mcL; platelets: 120-500 K/µL; hemoglobin 12.0-17.5 grams/dL); LFT's within 2x normal value; creatinine clearance test (CrCl) ≥ 50 mL/min; cholesterol (≤260 mg/dl), triglycerides≤ 400 mg/dl), and glucose control (HbA1c ≤ 8%). Prothrombin time/partial thromboplastin time/international normalized ratio (PT/PTT/INR) within normal limits
- Body mass index (BMI) within range of 19 - 35 kg/m2
- Must have a reliable informant or caregiver
- Participants must have no plans to travel that interfere with study visits
Exclusion Criteria
- Any medical or neurologic condition (other than Alzheimer's Disease) that might be a contributing cause of the subject's cognitive impairment
- Clinically significant unstable psychiatric illness in the past six months
- Significant hearing, vision, or motor deficits that interfere with participation
- Alcohol or drug abuse/dependence in the past six months
- Stroke, transient ischemic attack, or unexplained loss of consciousness in the past six months
- Unstable angina, myocardial infarction, advanced chronic heart failure, or clinically significant conduction abnormalities within the past six months
- Relevant brain hemorrhage, bleeding disorder and cerebrovascular abnormalities
- Diagnosis of HIV infection or AIDS (CD4 count 160, diastolic BP > 90 mmHg)
- Uncontrolled diabetes (HbA1c > 8%, or the current use of insulin)
- Significant systematic illness or infection in the past 30 days
- Pregnant women
Data sourced from ClinicalTrials.gov (NCT04552795). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.