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

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

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Trial summary

The objective of this study is to determine the safety and tolerability of fosgonimeton (ATH-1017) in subjects with mild to moderate Alzheimer's disease who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202. Read more about this study

Who is it for?: Ages 55 to 85 years
Conditions the study is for: Alzheimer Disease
What is being tested?: ATH-1017
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 423 participants
Where: 1 siteUnited States
Study dates: 30 Jun 2021to 23 Oct 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

Subject has completed the Week 26 visit of either of the two blinded parent studies (ATH-1017-AD-0201 or ATH-1017-AD-0202).
Reliable and capable support person/caregiver who is willing to accept responsibility for supervising the daily treatment or, if required, administering study drug.
Subject capable of giving signed informed consent, or by a legally acceptable representative.
Subjects must be in generally good health.
Male subjects and their partners must agree to continue to use a double-barrier method of contraception during the study, including the follow-up period, unless the partner is not of childbearing potential.

Who cannot join

Subject has experienced a serious adverse event during the parent study, which could present an increased safety risk during the open label extension.
New diagnosis of severe major depressive disorder even without psychotic features.
Any subject with formalized delusions or hallucinations.
Significant suicide risk.
Newly-diagnosed malignant tumor, except for the following conditions that are stable in the judgement of the investigator:
Adequately treated squamous and basal cell carcinoma, or squamous and basal cell carcinoma in situ
Prostate carcinoma in situ

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: Open-Label Extension of Studies ATH-1017-AD-0201 and ATH-1017-AD-0202 in Subjects With Mild to Moderate Alzheimer's Disease

Read the detailed description

This is a multicenter, seamless, open-label extension (OLEX) study of ATH-1017 treatment in subjects with a clinical diagnosis of mild to moderate Alzheimer's disease who completed 26 weeks treatment in the randomized, placebo-controlled, double-blind studies, ATH-1017-AD-0201 and ATH-1017-AD-0202. This OLEX study will provide additional, longer-term safety and tolerability information on ATH-1017 administration up to 48 months in subjects with mild to moderate Alzheimer's disease.

Conditions: Alzheimer Disease
Being tested: ATH-1017
Keywords: Alzheimer's, Dementia, ATH-1017, Open Label
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

Study groups in NCT04886063
Study group	Role	What they receive
Treatment	Experimental	Daily subcutaneous (SC) injection of ATH-1017 - 40mg Dosage Receives: Drug: ATH-1017

What is being measured

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

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Description - To determine the safety and tolerability of ATH-1017 in subjects with mild to moderate Alzheimer's disease (AD) who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202
Measured: Up to 173 weeks (study termination)

Timeline

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

30 Jun 202123 Oct 2024

First posted13 May 2021
Study start30 Jun 2021
Primary completion23 Oct 2024
Study completion23 Oct 2024

Why it stopped: ATH-1017-AD-0203 was terminated after the Ph2/3 parent trial (LIFT-AD; NCT04488419) did not meet its primary endpoint, the GST score.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Alzheimer DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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: Not applicable
Intervention model: Single group
Model description: Open label extension
Masking: None (open label)
Enrolment: 423 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04886063
Sponsor's protocol ID: ATH-1017-AD-0203
Status last verified: Mar 2025
First submitted: 10 May 2021
Last updated: 1 Apr 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Alzheimer Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 1 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Open-Label Extension of Studies ATH-1017-AD-0201 and ATH-1017-AD-0202 in Subjects With Mild to Moderate Alzheimer's Disease
ClinicalTrials.gov ID: NCT04886063
Acronym / sponsor ID: ATH-1017-AD-0203
Conditions: Alzheimer Disease
Keywords: Alzheimer's, Dementia, ATH-1017, Open Label
Phase: Phase 2/3
Study type: Interventional
Recruitment status: TerminatedReason stopped: ATH-1017-AD-0203 was terminated after the Ph2/3 parent trial (LIFT-AD; NCT04488419) did not meet its primary endpoint, the GST score.
Enrolment: 423 participants (actual)
Lead sponsor: LeonaBioIndustry (pharma, biotech, devices)
Study start: 30 Jun 2021 (actual)
Primary completion: 23 Oct 2024 (actual)
Study completion: 23 Oct 2024 (actual)
First posted: 13 May 2021 (actual)
Results: Posted 1 Apr 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Open label extension

Arm	Type	Receives
Treatment	Experimental	Drug: ATH-1017Daily subcutaneous (SC) injection of ATH-1017 - 40mg Dosage

Who may qualify

Ages: 55 to 85 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Subject has completed the Week 26 visit of either of the two blinded parent studies (ATH-1017-AD-0201 or ATH-1017-AD-0202).
Reliable and capable support person/caregiver who is willing to accept responsibility for supervising the daily treatment or, if required, administering study drug.
Subject capable of giving signed informed consent, or by a legally acceptable representative.
Subjects must be in generally good health.
Male subjects and their partners must agree to continue to use a double-barrier method of contraception during the study, including the follow-up period, unless the partner is not of childbearing potential.

Key exclusion criteria (7 listed)

Subject has experienced a serious adverse event during the parent study, which could present an increased safety risk during the open label extension.
New diagnosis of severe major depressive disorder even without psychotic features.
Any subject with formalized delusions or hallucinations.
Significant suicide risk.
Newly-diagnosed malignant tumor, except for the following conditions that are stable in the judgement of the investigator:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ATH-1017Daily subcutaneous (SC) injection of ATH-1017 in a pre-filled syringe	Drug	Treatment

Primary outcome

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: Up to 173 weeks (study termination)
Description - To determine the safety and tolerability of ATH-1017 in subjects with mild to moderate Alzheimer's disease (AD) who completed the 26-week randomized treatment in Study ATH-1017-AD-0201 or Study ATH-1017-AD-0202

Results and safety

Results posted to the registry on 1 Apr 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
ATH-1017 40 Milligrams (mg)	423	35

Primary outcome: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Time frame: Up to 173 weeks (study termination). Count of participants. Unit: Participants

Measure	ATH-1017 40 Milligrams (mg) n = 423
At least 1 TEAE	323
No TEAEs	100

Population analysed: Safety population: all participants who received at least one dose of study medication

Adverse events

Time frame: Up to 173 weeks; the protocolled 206-week treatment duration ended early due to study termination.. Other adverse events reported at 4% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
ATH-1017 40 Milligrams (mg)	54 of 423 (12.8%)	348 of 423 (82.3%)	4 of 423

Limitations and caveats (from the sponsor): The study was terminated early, following the completion of the parent study (ATH-1017-AD-0201; LIFT-AD)

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 38599894Reda 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.
Individual participant dataSharing planned: No

Related trials for Alzheimer Disease

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Active, not recruitingPhase 2
A Study to Evaluate the Safety and Biomarker Effects of RO7269162 in Participants at Risk for or at the Prodromal Stage of Alzheimer's Disease (AD)
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Recruiting
HEARS-NPS: Addressing Hearing Loss as a Common Unmet Contributor of Neuropsychiatric Symptoms
Alzheimer Disease, Dementia
RecruitingPhase 3
A Phase 3 Study to Evaluate the Safety and Efficacy of KarXT + KarX-EC for the Treatment of Agitation Associated With Alzheimer's Disease (ADAGIO-1)
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RecruitingObservational study
PET Imaging in the Baltimore Longitudinal Study of Aging (BLSA)
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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing ATH-1017 in people with Alzheimer Disease, 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 5 inclusion and 7 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: stopped early, as of the sponsor's last update on 1 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. 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 1 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?

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: Up to 173 weeks (study termination)). 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 NCT04886063, which also lists 1 linked publication.

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 1 Apr 2025.