MedPath

Innate Immunity Stimulation Via TLR9 in Early AD

Phase 1
Recruiting
Conditions
Mild Cognitive Impairment
Alzheimer Dementia
Interventions
Drug: Placebo
Registration Number
NCT05606341
Lead Sponsor
NYU Langone Health
Brief Summary

This single-center, double-blind, placebo-controlled study will recruit in total 39 participants with either Mild Cognitive Impairment due to Alzheimer's disease (MCI) or Mild Alzheimer's disease dementia (mild AD). There will be 3 Dose levels. An initial cohort of 13 subjects will be randomized to a Dose level 1 (0.1 mg/kg vs. placebo) lasting 8 weeks. An additional 13 subjects will be recruited and randomized into Dose level 2 (0.25 mg/kg vs. placebo) for 8 weeks and 13 subjects for the last Dose level 3 (0.5 mg/kg vs. placebo) for 8 weeks. The primary objective will be to assess safety and tolerability of CpG 1018.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
15
Inclusion Criteria
  1. 65-85 years of age
  2. MCI due to AD or mild AD dementia per NIA-AA specified criteria published in 2018
  3. Montreal Cognitive Assessment (MoCA) score ≥17 AND;
  4. Positive Florbetaben PET amyloid scan, or other positive PET amyloid scan performed within one year of study enrollment
  5. Must be able to provide consent or assent (If applicable).
  6. Must be willing and able to participate in all study related procedures.
  7. Must have a reliable study partner to provide information on the subject's cognitive and functional status. Study partner must have sufficient contact with the subject, as determined by the PI, and be available to accompany the subject to clinic visits or by phone.
Exclusion Criteria
  1. History of psychiatric illness (e.g. hallucinations, major depression, suicidal ideation or delusions) that could interfere with completion of study related procedures as determined by PI
  2. History of autoimmune disorders or antibody-mediated disease, severe asthma, or other serious infection or systemic illness, as determined by PI
  3. Use of corticosteroids or immunosuppressive drugs within 30 days of study entry
  4. History of splenectomy
  5. Renal impairment
  6. Use of chloroquine within 8 weeks of study entry
  7. Inability to undergo MRI imaging
  8. History of TIA, stroke or seizures within 12 months of screening
  9. Any neurological condition other than AD that could contribute to cognitive impairment (including related to possible "long COVID") as determined by PI
  10. Participation in any other current AD investigational interventional trial
  11. Current use of an anti-coagulant
  12. Current use of drugs that are major substrates of cytochrome P450 (CYP) enzyme 1A2
  13. Recent exposure to COVID-19 infection within 14 days or recent onset of symptoms within 14 days that may be related to COVID-19 infection

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo3 injections of sterile saline at Day 1, Week 4, and Week 8, followed by 1-hour post-dose observation period to check for injection site reaction and/or adverse reactions.
CpG 1018 0.25 mg/kgCpG10183 injections at Day 1, Week 4, and Week 8. Treatment administered as morning injection of dose 0.25 mg/kg, followed by 1-hour post-dose observation period to check for injection site reaction and/or adverse reactions.
CpG 1018 0.1 mg/kgCpG10183 injections at Day 1, Week 4, and Week 8. Treatment administered as morning injection of dose 0.1mg/kg, followed by 1-hour post-dose observation period to check for injection site reaction and/or adverse reactions.
CpG 1018 0.5 mg/kgCpG10183 injections at Day 1, Week 4, and Week 8. Treatment administered as morning injection of dose 0.5 mg/kg, followed by 1-hour post-dose observation period to check for injection site reaction and/or adverse reactions.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants with Antineutrophil Cytoplasmic Antibody (ANCA) Confirmed by Autoimmunity Marker Screening Test ResultUp to Week 18

Evaluation of ANCA in patient blood samples at Baseline, Day 56, Week 14 and Week 18.

Percentage of Participants with Amyloid-Related Imaging Abnormalities-Edema (ARIA-E) Confirmed by Magnetic Resonance Imaging (MRI)Up to Week 14

Evaluation of ARIA-E at Baseline and Week 14 using 3T PET/MR Siemens Biograph system.

Percentage of Participants with Rheumatoid Factor (RF) Confirmed by Autoimmunity Marker Screening Test ResultUp to Week 18

Evaluation of RF in patient blood samples at Baseline, Day 56, Week 14 and Week 18.

Percentage of Participants with Antinuclear Antibody (ANA) Confirmed by Autoimmunity Marker Screening Test ResultUp to Week 18

Evaluation of ANA in patient blood samples at Baseline, Day 56, Week 14 and Week 18.

Number of Patient-Reported Adverse Events (AEs)Up to Week 18

AEs defined as any symptom, sign, illness or experience that develops or worsens in severity during the course of the study.

Percentage of Participants with Amyloid-Related Imaging Abnormalities-Haemosiderin (ARIA-H) Confirmed by Magnetic Resonance Imaging (MRI)Up to Week 14

Evaluation of ARIA-H at Baseline and Week 14 using 3T PET/MR Siemens Biograph system.

Secondary Outcome Measures
NameTimeMethod
Change in CSF Tau Biomarker ConcentrationBaseline, Week 18

Tau biomarker concentration detected via CSF analysis.

Change in AD Assessment Scale Cognitive Subscale (ADAS-Cog-13) ScoresBaseline, Week 18

13-item self-assessment measuring levels of cognitive and non-cognitive dysfunctions from mild to severe. Total scores range from 0 to 85. Lower scores indicate greater cognitive performance. A decrease in scores indicates cognitive performance improved during the observational period.

Change in AD Cooperative Study-Activities of Daily Living Inventory, Mild Cognitive Impairment version (ADCS-ADL-MCI) ScoresBaseline, Week 18

18-item questionnaire measuring a participant's basic and instrumental activities of daily living over the previous month. Total scores range from 0-53, where higher scores indicate greater competence in performing activities. An increase in scores indicates competence increased during the observational period.

Change in Montreal Cognitive Assessment (MoCa) ScoreBaseline, Week 18

30-item assessment of global cognitive function. Total scores range from 0 to 30, with higher scores indicating greater cognitive function. Scores of 26 and higher are consider to be normal. An increase in scores indicates cognitive function increased during the observational period.

Change in Plasma Amyloid Biomarker ConcentrationBaseline, Week 18

Amyloid biomarker concentration detected via plasma analysis.

Change in Columbia-Suicide Severity Rating Scale (C-SSRS) ScoresBaseline, Week 18

C-SSRS systematically tracks suicidal ideation and behavior. The total score range is 0 (no ideation is present) to 5 (active suicidal ideation with specific plan and intent). A decrease in scores indicates suicidal ideation and behavior decreased during the observational period.

Change in Global Clinical Dementia Rating (CDR-Global)Baseline, Week 18

5-point questionnaire assessing six domains of cognitive and functional performance applicable to Alzheimer's disease and related dementias: Memory, Orientation; Judgement \& Problem Solving; Community Affairs; Home \& Hobbies; and Personal Care. Higher scores indicate greater severity of dementia: 0= Normal, 0.5=very mild dementia, 1=mild dementia, 2=moderate dementia, 3=severe dementia.

Change in Cerebral Spinal Fluid (CSF) Amyloid Biomarker ConcentrationBaseline, Week 18

Amyloid biomarker concentration detected via CSF analysis.

Change in Plasma Tau Biomarker ConcentrationBaseline, Week 18

Tau biomarker concentration detected via plasma analysis.

Trial Locations

Locations (1)

NYU Langone Health

🇺🇸

New York, New York, United States

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