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Alzheimer DiseasePhase 2/3LIFT-ADNCT04488419

ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease

A Phase 2/3 study testing ATH-1017 in people with Alzheimer Disease and Dementia of Alzheimer Type, run by LeonaBio at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study is designed to evaluate safety and efficacy of fosgonimeton (ATH-1017) in the treatment of mild to moderate Alzheimer's disease with a randomized treatment duration of 26-weeks. Read more about this study

Who is it for?
Ages 55 to 85 years
Conditions the study is for
Alzheimer Disease, Dementia of Alzheimer Type
What is being tested?
ATH-1017
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
554 participants
Where
1 siteUnited States
Study dates
28 Sep 2020to 15 Jul 2024
Phase
Phase 2/3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age 55 to 85 years
  • Mild-to-moderate AD dementia subjects, MMSE 14-24, CDR 1 or 2 at Screening
  • Clinical diagnosis of dementia, due probably to AD, by Revised National Institute on Aging-Alzheimer's Association criteria (McKhann, 2011)
  • Body mass index (BMI) of ≥ 18 and ≤ 35 kg/m2 at Screening
  • Reliable and capable support person/caregiver
  • Treatment-free (subjects not receiving acetylcholinesterase inhibitor [AChEI] treatment), defined as:
  • Treatment-naïve, OR
  • Subjects who received an AChEI in the past and discontinued at least 4 weeks prior to Screening

Who cannot join

  • History of significant neurologic disease, other than AD, that may affect cognition, or concurrent with the onset of dementia
  • Subject has atypical variant presentation of AD, if known from medical history, particularly non-amnestic AD
  • History of brain MRI scan indicative of any other significant abnormality
  • Diagnosis of severe major depressive disorder even without psychotic features.
  • Significant suicide risk
  • History within 2 years of Screening, or current diagnosis of psychosis
Show all 16Show fewer
  • Myocardial infarction or unstable angina within the last 6 months
  • Clinically significant cardiac arrhythmia (including atrial fibrillation), cardiomyopathy, or cardiac conduction defect (note: pacemaker is acceptable)
  • Subject has either hypertension or symptomatic hypotension
  • Clinically significant ECG abnormality at Screening
  • Chronic kidney disease with estimated glomerular filtration rate (eGFR) <45 mL/min
  • Hepatic impairment with alanine aminotransferase or aspartate aminotransferase > 2 times the upper limit of normal, or Child-Pugh class B and C
  • Malignant tumor within 3 years before Screening
  • Memantine in any form, combination or dosage within 4 weeks prior to Screening
  • Acetylcholinesterase inhibitors in any dosage form
  • The subject has received active amyloid or tau immunization (i.e., vaccination for Alzheimer's disease) at any time, or passive immunization (i.e., monoclonal antibodies for Alzheimer's disease) within 6 months of Screening

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Rochester-AD-CARE Program

    Rochester, New York, 14620

Other trials in United States

About this study

Official title: A Randomized, Placebo-Controlled, Double-Blind Study of ATH-1017 Treatment in Subjects With Mild to Moderate Alzheimer's Disease

This study is designed to evaluate safety and efficacy of fosgonimeton (ATH-1017) in the treatment of mild to moderate Alzheimer's disease with a randomized treatment duration of 26-weeks.

Read the detailed description

The study is designed to evaluate safety and efficacy of ATH-1017 in mild to moderate AD subjects, with randomized, parallel-arm treatment duration of 26 weeks, and based on clinical diagnostic criteria of Alzheimer's disease. Clinical efficacy is demonstrated by improvement in cognition and global/functional assessments comparing treatment to placebo.

Being tested
ATH-1017, Placebo
Keywords
Alzheimer's Disease, Cognition, Dementia, ATH-1017, LIFT AD, Fosgonimeton
Medical subject headings
Alzheimer Disease, Dementia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ATH-1017Drug

    Daily subcutaneous (SC) injection of ATH-1017 in a pre-filled syringe

  • PlaceboDrug

    Daily subcutaneous (SC) injection of Placebo in a pre-filled syringe

Study groups in NCT04488419
Study groupRoleWhat they receive
DosageExperimentalDaily subcutaneous (SC) injection of 40mg ATH-1017
Receives: Drug: ATH-1017
PlaceboPlacebo comparatorDaily subcutaneous (SC) injection of Placebo
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Global Statistical Test (GST) Score

    The Global Statistical Test (GST) score is a composite of cognition and function, calculated as the average of two change from baseline z-scores; the z-scores are calculated for the change from baseline scores for cognition (Alzheimer's Disease Assessment Scale-Cognitive Subscale [ADAS-Cog11]; lower value indicates improvement) and function (Alzheimer's Disease Cooperative Study - Activities of Daily Living, 23-item version [ADCS-ADL23] score; higher value indicates improvement).

    GST is a standardized score relative to the population mean. Therefore, a GST score of 0 is representative of the population mean. Since GST is a composite of two endpoints, a negative ADCS-ADL23 score is used in deriving GST. Therefore, a lower score indicates improvement, and a higher score indicates worsening. There are no defined clinically relevant thresholds for this GST score.

    Measured: Baseline and Week 26

Secondary outcomes (3)

  1. Alzheimer's Disease Assessment Scale-Cognitive Subscale 11 (ADAS-Cog11) Change From Baseline

    The ADAS-Cog11 was designed to measure cognitive symptom change in participants with Alzheimer's Disease (AD) and consisted of 11 tasks. The standard 11 items (and corresponding score range) were: word recall (0-10), commands (0-5), constructional praxis (0-5), naming objects and fingers (0-5), ideational praxis (0-5), orientation (0-8), word recognition (0-12), spoken language ability (0-5), comprehension of spoken language (0-5), word-finding difficulty (0-5), and remembering test instructions (0-5). The test included 7 performance items and 4 clinician-rated items. The ADAS-Cog11 total score was the sum of all 11 individual items, with a total score ranging from 0 (no impairment) to 70 (severe impairment). Higher scores indicated more severe cognitive impairment. Baseline was defined as Day 1.

    Measured: Baseline and Week 26

  2. Alzheimer's Disease Cooperative Study - Activities of Daily Living, 23-Item Version (ADCS-ADL23) Change From Baseline

    The ADCS-ADL23 is a 23-item assessment of functional impairment in terms of activities of daily living administered to the support person/caregiver. It comprises 23 questions about the subject's involvement and level of performance across items representing daily living. The questions range from basic to instrumental activities of daily living. Each item is rated from the highest level of independent performance to complete loss. The total score range is from 0 to 78, with lower scores indicating greater functional impairment. Baseline was defined as Day 1.

    Measured: Baseline and Week 26

  3. Plasma Neurofilament Light Chain (NfL) Concentrations Change From Baseline

    Neurofilament light chain (NfL) is an objective biomarker of neurodegeneration and was measured to quantitatively support the evaluation of ATH-1017. Higher concentrations of NfL in plasma are associated with neurodegeneration.

    Measured: Baseline and Week 26

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Jul 2020
  2. Study start28 Sep 2020
  3. Primary completion15 Jul 2024
  4. Study completion15 Jul 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Randomized, double-blind, placebo-controlled, parallel-group study
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
554 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04488419
Sponsor's protocol ID
ATH-1017-AD-0201
Acronym
LIFT-AD
Status last verified
Apr 2025
First submitted
23 Jul 2020
Last updated
4 Apr 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • McKhann GM, Knopman DS, Chertkow H, Hyman BT, Jack CR Jr, Kawas CH, Klunk WE, Koroshetz WJ, Manly JJ, Mayeux R, Mohs RC, Morris JC, Rossor MN, Scheltens P, Carrillo MC, Thies B, Weintraub S, Phelps CH. The diagnosis of dementia due to Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease. Alzheimers Dement. 2011 May;7(3):263-9. doi: 10.1016/j.jalz.2011.03.005. Epub 2011 Apr 21.

    PubMed 21514250

  • Reda SM, Setti SE, Berthiaume AA, Wu W, Taylor RW, Johnston JL, Stein LR, Moebius HJ, Church KJ. Fosgonimeton attenuates amyloid-beta toxicity in preclinical models of Alzheimer's disease. Neurotherapeutics. 2024 Jul;21(4):e00350. doi: 10.1016/j.neurot.2024.e00350. Epub 2024 Apr 9.

    PubMed 38599894

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing ATH-1017 in people with Alzheimer Disease and Dementia of Alzheimer Type, run by LeonaBio at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Global Statistical Test (GST) Score (time frame: Baseline and Week 26). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04488419, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Apr 2025; AzTrials last refreshed its copy on 8 Nov 2026.

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