A Phase II/III Multicenter Randomized, Double-Blind, Placebo-Controlled Platform Trial of Potential Disease Modifying Therapies Utilizing Biomarker, Cognitive, and Clinical Endpoints in Dominantly Inherited Alzheimer's Disease

Trial for genetic Alzheimer's: testing new treatments to slow disease

NCT: NCT05269394 · Status: ACTIVE NOT RECRUITING · Phase: Phase 3 · Sponsor: Washington University School of Medicine · Started: 2021-12-22 · Est. Completion: 2028-07

Plain English Summary

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU) is a Phase 3 clinical trial sponsored by Washington University School of Medicine studying Alzheimers Disease, Dementia, Alzheimers Disease, Familial. This trial tests new medications (E2814 and Lecanemab) to see if they can slow down the progression of Alzheimer's disease in people with a specific genetic mutation. It is for individuals aged 18-80 who have a known gene mutation that causes early-onset Alzheimer's and are showing early signs of cognitive changes or are at risk. Participation involves regular visits for tests like brain scans (MRI, PET), spinal fluid collection (lumbar puncture), cognitive assessments, and receiving study medication or a placebo. Currently, there are no approved treatments that can stop or reverse the progression of this specific type of inherited Alzheimer's disease. The trial aims to enroll 197 participants.

Official Summary

To assess the safety, tolerability, biomarker, cognitive, and clinical efficacy of investigational products in participants with an Alzheimer's disease-causing mutation by determining if treatment with the study drug improves disease-related biomarkers and slows the rate of progression of cognitive or clinical impairment.

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you are between 18 and 80 years old and have a confirmed gene mutation that causes early-onset Alzheimer's. You should be within 10 years of when symptoms are expected or started, and have normal cognition, mild cognitive impairment, or mild dementia. You cannot join if you have other significant brain or psychiatric conditions, a history of recent suicidal thoughts, or certain medical conditions that would prevent MRI scans or affect your health. You must be able to undergo all required tests, including MRI and spinal taps, and have a reliable study partner to help provide information. This trial is studying Alzheimers Disease, Dementia, Alzheimers Disease, Familial, so participants generally need a confirmed diagnosis.

What They're Measuring

The primary outcome measures how the treatment affects tau protein buildup in the brain, which is a key indicator of Alzheimer's progression, aiming to show if the drug can slow this process. The specific primary outcome measures are: The primary end point is the change from Week 24 to Week 104 and Week 208 in tau PET in the Symptomatic Population (Cohort 1). (Weeks 24, 104, and 208). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial is important because it addresses a critical unmet need for treatments that can modify the course of dominantly inherited Alzheimer's disease, a form caused by specific genetic mutations. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Alzheimers Disease, Dementia, Alzheimers Disease, Familial, where improved treatment options are needed.

Investor Insight

This trial targets a specific, rare form of Alzheimer's, but success could pave the way for broader Alzheimer's treatments, making it a significant area for investment in the competitive neurodegenera Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor about the specific genetic mutation you have and how it relates to this trial. Understand that participation requires commitment to multiple visits over several years for various medical tests and assessments. Be prepared for potential side effects and the possibility of receiving a placebo instead of an active drug. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

Interventions

Primary Outcomes

Secondary Outcomes

Full Eligibility Criteria

Key Inclusion Criteria:

* Between 18-80 years of age
* Individuals who know they have an Alzheimer's disease-causing mutation.
* Are within -10 to + 10 years of the predicted or actual age of cognitive symptom onset.
* Cognitively normal or with mild cognitive impairment or mild dementia, Clinical Dementia Rating (CDR) of 0-1 (inclusive)
* Fluency in DIAN-TU trial approved language and evidence of adequate premorbid intellectual functioning
* Able to undergo Magnetic Resonance Imaging (MRI), Lumbar Puncture (LP), Positron Emission Tomography (PET), and complete all study related testing and evaluations.
* For women of childbearing potential, if partner is not sterilized, participant must agree to use effective contraceptive measures (hormonal contraception, intra-uterine device, sexual abstinence, barrier method with spermicide).
* Adequate visual and auditory abilities to perform all aspects of the cognitive and functional assessments.
* Has a Study Partner who in the investigator's judgment is able to provide accurate information as to the subject's cognitive and functional abilities, who agrees to provide information at the study visits which require informant input for scale completion.

Key Exclusion Criteria:

* Significant neurologic disease (other than AD) or psychiatric disease that may currently or during the course of the study affect cognition or participant's ability to complete the study.
* At high risk for suicide, e.g., significant suicidal ideation or attempt within last 12 months. Current stable mild depression or current use of antidepressant medications is not exclusionary.
* History or presence of brain MRI scans indicative of any other significant abnormality
* Substance or alcohol use disorder currently or within the past 1 year
* Presence of pacemakers, aneurysm clips, artificial heart valves, ear implants, or foreign metal objects in the eyes, skin or body which would preclude MRI scan.
* History or presence of clinically significant cardiovascular disease, hepatic/renal disorders, infectious disease or immune disorder, or metabolic/endocrine disorders
* Anticoagulants except low dose (≤ 325 mg) aspirin.
* Have been exposed to a monoclonal antibody targeting beta amyloid peptide within the past six months.
* History of cancer within the last 5 years, except basal cell carcinoma, non-squamous skin carcinoma, prostate cancer or carcinoma in situ with no significant progression over the past 2 years.
* Positive urine or serum pregnancy test or plans or desires to become pregnant during the course of the trial.
* Subjects unable to complete all study related testing, including implanted metal that cannot be removed for MRI scanning, required anticoagulation and pregnancy.

Trial Locations

Frequently Asked Questions

What is clinical trial NCT05269394?

NCT05269394 is a Phase 3 INTERVENTIONAL study titled "Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia. A Study of Potential Disease Modifying Treatments in Individuals With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU)." It is currently active, not recruiting and is sponsored by Washington University School of Medicine. The trial targets enrollment of 197 participants.

What conditions does NCT05269394 study?

This trial investigates treatments for Alzheimers Disease, Dementia, Alzheimers Disease, Familial. The primary condition under study is Alzheimers Disease.

What treatments are being tested in NCT05269394?

The interventions being studied include: E2814 (DRUG), Lecanemab (DRUG), Matching Placebo (E2814) (DRUG). Administered intravenously in a blinded fashion

What does Phase 3 mean for NCT05269394?

Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.

What is the current status of NCT05269394?

This trial is currently "Active, Not Recruiting." It started on 2021-12-22. The estimated completion date is 2028-07.

Who is sponsoring NCT05269394?

NCT05269394 is sponsored by Washington University School of Medicine. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT05269394?

The trial aims to enroll 197 participants. The trial status is active, not recruiting.

How is NCT05269394 designed?

This is a interventional study, uses randomized allocation, follows a parallel design, employs quadruple masking. Masking means some participants and/or investigators do not know which treatment group a participant is in, which helps reduce bias.

What are the primary outcomes being measured in NCT05269394?

The primary outcome measures are: The primary end point is the change from Week 24 to Week 104 and Week 208 in tau PET in the Symptomatic Population (Cohort 1). (Weeks 24, 104, and 208). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT05269394 being conducted?

This trial is being conducted at 20 sites, including Birmingham, Alabama; La Jolla, California; Los Angeles, California; New Haven, Connecticut and 16 more sites (United States, Argentina, Australia).

Where can I find official information about NCT05269394?

The official record for NCT05269394 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT05269394. This government database provides the most up-to-date and detailed information about the trial.

What is NCT05269394 testing in simple terms?

This trial tests new medications (E2814 and Lecanemab) to see if they can slow down the progression of Alzheimer's disease in people with a specific genetic mutation. It is for individuals aged 18-80 who have a known gene mutation that causes early-onset Alzheimer's and are showing early signs of cognitive changes or are at risk.

Why is this trial significant?

This trial is important because it addresses a critical unmet need for treatments that can modify the course of dominantly inherited Alzheimer's disease, a form caused by specific genetic mutations. As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.

What are the potential risks of participating in NCT05269394?

Common side effects may include reactions at the injection site, headache, and fatigue. More serious risks, though rare, can include brain swelling or bleeding, especially with certain types of Alzheimer's medications. The study involves procedures like lumbar punctures and MRI scans, which carry their own small risks. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT05269394?

Ask your doctor about the specific genetic mutation you have and how it relates to this trial. Understand that participation requires commitment to multiple visits over several years for various medical tests and assessments. Be prepared for potential side effects and the possibility of receiving a placebo instead of an active drug. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT05269394 signal from an investment perspective?

This trial targets a specific, rare form of Alzheimer's, but success could pave the way for broader Alzheimer's treatments, making it a significant area for investment in the competitive neurodegenera This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.

What happens if the treatment in this trial doesn't work?

Participation involves regular visits for tests like brain scans (MRI, PET), spinal fluid collection (lumbar puncture), cognitive assessments, and receiving study medication or a placebo. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.