---
title: "Open Label Study of ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease"
nct_id: NCT04886063
overall_status: TERMINATED
phase: PHASE2, PHASE3
sponsor: LeonaBio
study_type: INTERVENTIONAL
primary_condition: Alzheimer Disease
countries: United States
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04886063.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04886063"
ct_last_update_post_date: 2025-04-01
last_seen_at: "2026-05-12T06:38:09.285Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Open Label Study of ATH-1017 for Treatment of Mild to Moderate Alzheimer's Disease

**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

**NCT ID:** [NCT04886063](https://clinicaltrials.gov/study/NCT04886063)

## Key Facts

- **Status:** TERMINATED
- **Why 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.
- **Phase:** PHASE2, PHASE3
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 423
- **Lead Sponsor:** LeonaBio
- **Conditions:** Alzheimer Disease
- **Start Date:** 2021-06-30
- **Completion Date:** 2024-10-23
- **CT.gov Last Update:** 2025-04-01

## Brief 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.

## 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.

## Eligibility

- **Minimum age:** 55 Years
- **Maximum age:** 85 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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
```

## Arms

- **Treatment** (EXPERIMENTAL) — Daily subcutaneous (SC) injection of ATH-1017 - 40mg Dosage

## Interventions

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

## Primary Outcomes

- **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

## Locations (1)

- University of Rochester-AD-CARE Program, Rochester, New York, United States

## Recent Field Changes (last 30 days)

- `locations.university of rochester-ad-care program|rochester|new york|united states` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.whyStopped` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04886063.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04886063*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*