ALZN002 for Alzheimer's Disease

Overseen By Donald P Reitberg, PharmD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Alzamend Neuro, Inc.

Must not be taking: Anticholinergics, Antidepressants, Antipsychotics, others

Disqualifiers: Schizophrenia, Bipolar, Stroke, others

Compensation: Varies

Number of Visits: Unknown

Duration: 21 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ALZN002 for individuals with mild to moderate Alzheimer's disease. Researchers aim to determine if this treatment can reduce symptoms by targeting amyloid beta, a protein linked to Alzheimer's. Participants will be randomly assigned to receive either ALZN002 or a placebo (a harmless substance with no therapeutic effect) to compare outcomes. Those with a confirmed diagnosis of Alzheimer's and ongoing memory issues may be suitable for this study. As a Phase 1, Phase 2 trial, the study focuses on understanding how ALZN002 works in people and measuring its effectiveness in an initial group, offering participants a chance to contribute to groundbreaking research.

Will I have to stop taking my current medications?

The trial does not specify if you must stop all current medications, but certain medications are restricted. You must have a stable dose of cholinesterase inhibitors or memantine for at least 60 days before the trial and maintain it during the study. Some medications like certain blood thinners and sleep aids are allowed under specific conditions.

Is there any evidence suggesting that ALZN002 is likely to be safe for humans?

Research shows that ALZN002 is a treatment under testing for Alzheimer's disease. In similar studies, a related treatment called AL-108 was generally safe and well-tolerated by people with mild memory problems over 12 weeks. This suggests that ALZN002 might also be safe for humans, but testing continues. Since ALZN002 is in early trials, researchers focus primarily on its safety. They carefully observe how the body responds to the treatment and monitor for any side effects. Joining a trial helps gather important information on how well people can handle the treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Alzheimer's?

Most treatments for Alzheimer's disease, like donepezil and memantine, aim to manage symptoms by modulating neurotransmitter activity. However, ALZN002 works differently by targeting the immune system. This treatment uses autologous dendritic cells (DCs) that are primed with a mutant peptide, potentially altering the immune response to slow or halt disease progression. Researchers are excited about ALZN002 because it represents a novel mechanism of action that could offer a new way to tackle Alzheimer's beyond just symptom management.

What evidence suggests that ALZN002 might be an effective treatment for Alzheimer's?

Research shows that ALZN002, a treatment for Alzheimer's disease under study in this trial, appears promising based on animal studies. In these studies, ALZN002 significantly reduced amyloid plaques, protein buildups in the brain linked to Alzheimer's. Although this data comes from animal studies, it provides a strong basis for its potential in humans. Additionally, other studies have shown that similar treatments are safe for people with Alzheimer's-related conditions. This supports the idea that ALZN002 could help reduce symptoms or slow the disease's progression. Participants in this trial will receive either ALZN002 or a placebo to evaluate its effectiveness and safety. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for individuals aged 60-85 with mild-to-moderate Alzheimer's dementia, who can consent and adhere to study requirements. They must have a confirmed diagnosis of Alzheimer's via PET scan, be willing to undergo further PET scans and MRIs, use contraception if necessary, and have a reliable study partner. Excluded are those with prior immunotherapies for Alzheimer's or other conditions, non-Alzheimer’s dementia or cognitive impairments, major psychiatric disorders, autoimmune diseases, current malignancy or infectious diseases.

Inclusion Criteria

I am willing and able to undergo MRI scans at specified times after my treatment doses.

I agree to use barrier contraception during and up to 30 days after the study.

I am willing and able to undergo a procedure to collect white blood cells.

Exclusion Criteria

Positive urine drug screen for controlled substances, alcohol abuse, or any condition that may unfavorably alter the risk-benefit of subject participation

I am not taking medications that could affect my thinking or blood clotting.

I have previously received immunotherapies targeting Aβ.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple ascending doses of ALZN002 or placebo to assess safety and tolerability

21 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

What Are the Treatments Tested in This Trial?

Interventions

• ALZN002

Trial Overview The ALZN002-01 trial tests the safety and effectiveness of ALZN002 (a personalized cell therapy using one’s own immune cells modified with an amyloid beta mutant peptide) against placebo in people with mild-to-moderate Alzheimer’s dementia. Participants will be randomly assigned to receive either the experimental treatment or placebo in a double-blind setup where neither they nor the researchers know who gets what.

How Is the Trial Designed?

Group I: Active Experimental Treatment 1 Intervention

ALZN002 (autologous DCs pulsed with E22W mutant peptide).

Group II: Placebo Placebo Group 1 Intervention

Saline ID and IV administrations.

Who Is Running the Clinical Trial?

Alzamend Neuro, Inc.

Lead Sponsor

Trials

2

Recruited

100+

bioRASI, LLC

Industry Sponsor

Trials

14

Recruited

3,400+

Published Research Related to This Trial

Alzhemed (tramiprosate) is a well-tolerated treatment for mild-to-moderate Alzheimer's disease that effectively reduces levels of the neurotoxic amyloid-beta peptide in the cerebrospinal fluid after 3 months of treatment.

While Alzhemed did not show immediate cognitive improvements after 3 months, long-term follow-up suggested stabilization of cognitive function, particularly in patients with mild Alzheimer's disease, indicating potential benefits over extended use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Alzhemed: a potential treatment for Alzheimer's disease. Aisen, PS., Gauthier, S., Vellas, B., et al.[2019]

Akatinol memantine significantly improved cognitive function, behavior, and mood in patients with mild to moderate Alzheimer's disease, showing similar efficacy to donepezil over a 16-week treatment period with 100 participants.

The treatment was found to be safe, with only mild and transient adverse events reported in 6% of patients, indicating that akatinol memantine is a viable option for managing Alzheimer's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical efficacy and safety of akatinol memantine in treatment of mild to moderate Alzheimer disease: a donepezil-controlled, randomized trial]. Hu, HT., Zhang, ZX., Yao, JL., et al.[2018]

In a review of safety data from five 18-month Alzheimer's disease trials, common adverse events included dyspnea (5.3%-5.8%), headache (4.0%-5.5%), and constipation (4.3%-4.7%), indicating a consistent profile of side effects among patients taking placebo.

Larger multinational studies showed higher overall discontinuation rates (24.6%-33.0%) compared to smaller studies (8.2%-21.0%), suggesting that study size and geography may influence patient retention and safety outcomes in Alzheimer's trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety profile of Alzheimer's disease populations in Alzheimer's Disease Neuroimaging Initiative and other 18-month studies. Henley, DB., Sundell, KL., Sethuraman, G., et al.[2021]

In an 11-week study involving 181 patients with mild to moderate Alzheimer's disease, the alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid modulator LY451395 did not show significant improvement in cognitive function compared to placebo, as measured by the ADAS-Cog scale.

While there were some marginal improvements in neuropsychiatric symptoms after 8 weeks of treatment, the overall safety profile was similar between LY451395 and placebo, with most adverse events being mild.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

AMPA potentiator treatment of cognitive deficits in Alzheimer disease. Chappell, AS., Gonzales, C., Williams, J., et al.[2022]

In a study involving 144 subjects with amnestic mild cognitive impairment, AL-108 was found to be generally safe and well tolerated over 12 weeks of treatment.

While the overall efficacy analysis did not show a significant difference in cognitive memory scores between AL-108 and placebo, there were promising signals of potential efficacy in specific memory and attention tasks, suggesting further research is warranted.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A double-blind, placebo-controlled, ascending-dose, randomized study to evaluate the safety, tolerability and effects on cognition of AL-108 after 12 weeks of intranasal administration in subjects with mild cognitive impairment. Morimoto, BH., Schmechel, D., Hirman, J., et al.[2016]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05834296

NCT05834296 | Study in Subjects With Mild-to-Moderate ... The primary purpose of this study is to assess the safety and tolerability of multiple ascending doses of ALZN002 compared with that of placebo in subjects ...

2. ir.alzamend.com ir.alzamend.com/news-events/press-releases/detail/53/alzamend-neuro-announces-the-initiation-of-a-phase-iiia

Press Releases Strong evidence of significant ALZN002‑mediated amyloid plaque reductions was observed in mouse disease models. There were no undue adverse ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10338203/

a randomised, double-blind, placebo-controlled, phase 2a study This phase 2a study met its primary prespecified outcomes and indicated the safety of active immunotherapy with UB-311 in amyloid PET-positive ...

4. labiotech.eu labiotech.eu/trends-news/alzamend-alzheimers-vaccine-trial/

Alzamend pushing ahead with Alzheimer's vaccine trial Strong evidence of significant ALZN002‑mediated amyloid plaque reductions was observed in mouse disease models. ... NodThera announces positive ...

5. withpower.com withpower.com/trial/phase-2-alzheimer-disease-4-2023-eb1c3

ALZN002 for Alzheimer's Disease · Info for Participants In a study involving 144 subjects with amnestic mild cognitive impairment, AL-108 was found to be generally safe and well tolerated over 12 weeks of treatment.

6. ir.alzamend.com ir.alzamend.com/news-events/press-releases/detail/48/alzamend-neuro-partners-with-biorasi-to-conduct-a

Alzamend Neuro Partners With Biorasi to Conduct a First-in ... The purpose of this trial will be to assess the safety, tolerability, and efficacy of multiple ascending doses of ALZN002 compared with that of placebo.

7. alzamend.com alzamend.com/pipeline/

Product Pipeline ALZN002, is a patented method using a mutant-peptide sensitized cell as a cell-based therapeutic vaccine which seeks to restore the ability of the patient's ...

8. firstwordpharma.com firstwordpharma.com/story/5690802

Alzamend Neuro Partners With the Miller School of ... These DCs are pulsed with a novel amyloid-beta peptide (E22W) designed to bolster the ability of the patient's immune system to combat Alzheimer's; the goal of ...