Study of ASN51 in Adults With Early Alzheimer's Disease

NCT06677203 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: ASN51-201
• Study Type: interventional
• Target: 123
• Locations: 1 country
• Sites: 7

Brief Summary

The main purpose of this study is to evaluate the safety, tolerability, and effect on biomarkers of disease pathophysiology and pathology, pharmacokinetics (PK), and preliminary effects on measures of clinical efficacy of multiple doses of ASN51 in adult participants with early Alzheimer's disease (AD).

Conditions

Alzheimer Disease

Keywords

Alzheimer's Disease; ASN51

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Adverse Events (AEs)

  An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related.

  From first dose up to end of the study up to Week 28

• Change From Baseline in Columbia-Suicide Severity Rating Scale (C-SSRS)

  C-SSRS is used to assess the suicidality of participants and assessment includes "yes" or "no" responses for 5 questions, each related to suicidal ideation and suicidal behavior. Numeric ratings are provided for suicidal ideation (score ranges from 1 to 5, where higher scores indicate more suicidal ideation) and suicidal behavior (score ranges from 0 to 4 where higher total scores indicate more suicidal behavior).

  Baseline up to Week 28

Secondary Outcomes (6)

• Change From Baseline in Cerebrospinal Fluid (CSF) Plasma Tau Phosphorylated at Threonine-217 (pTau217) Through Week 24

  Baseline through Week 24

• Change From Baseline in CSF Total Tau Protein Through Week 24

  Baseline through Week 24

• Change From Baseline in Plasma pTau217 Through Week 24

  Baseline through Week 24

• Change From Baseline in MK-6240 Tau Positron Emission Tomography (PET) Signal Through Week 24

  Baseline through Week 24

• Trough Plasma Concentration (Cmin) of ASN51 in Plasma at Steady State

  Pre-dose on Day 1 and at multiple time points post-dose up to Week 24

Study Arms (3)

ASN51: Low Dose

EXPERIMENTAL

Participants were to receive low dose of ASN51 orally, once daily (QD) for up to 24 weeks in the double-blind placebo-controlled intervention period or up to 36 weeks long-term extension period.

Drug: ASN51

ASN51: High Dose

EXPERIMENTAL

Participants were to receive high dose of ASN51 orally, QD for up to 24 weeks in the double-blind placebo-controlled intervention period or up to 36 weeks long-term extension period.

Drug: ASN51

Placebo

PLACEBO COMPARATOR

Participants were to receive ASN51 matching placebo orally, QD for up to 24 weeks in the double-blind placebo-controlled intervention period.

Drug: Placebo

Interventions

ASN51 DRUG

Oral capsules

ASN51: High Dose; ASN51: Low Dose

Placebo DRUG

Oral capsules

Placebo

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female age 50 to 80 years.
• A clinical diagnosis of Alzheimer's disease (AD) at either the mild cognitive impairment or mild AD dementia stage per National Institute on Aging and the Alzheimer's Association, consistent with Stage 3 and Stage 4 in the Food and Drug Administration (FDA) draft guidance for early AD.
• Mini-Mental State Examination score of 20 to 28 (inclusive).
• A plasma pTau217 result consistent with the presence of amyloid pathology.
• Must have a care partner who, in the investigator's judgment, has frequent and sufficient contact with the participant as to be able to provide accurate information about the participant's cognitive and functional abilities. The care partner must be literate and provide informed consent.

You may not qualify if:

• Any medical or neurological/neurodegenerative condition (other than AD) that, in the opinion of the Investigator, might be a contributing cause to the participant's cognitive impairment (e.g., current history of substance abuse, uncontrolled vitamin B12 deficiency or abnormal thyroid function, stroke or other cerebrovascular condition, normal pressure hydrocephalus, Parkinson's Disease, Lewy body dementia, cerebral amyloid angiopathy, frontotemporal dementia) or could lead to discontinuation, lack of compliance, interference with study assessments, or safety concerns.
• Non-amnestic presentation of AD as judged by the investigator.
• Woman of childbearing potential.
• Any prior or ongoing exposure to active or passive anti-amyloid immunotherapy, anti-tau immunotherapy, an anti-tau antisense oligonucleotide or gene therapy, or O-linked-β-N-acetylglucosaminidase (O-GlcNAcase) inhibitor.

Sponsors & Collaborators

• Asceneuron S.A.: lead

Study Sites (7)

K2 Medical Research

Clermont, Florida, 34711, United States

Location

K2 Medical Research - The Villages

Lady Lake, Florida, 32159, United States

Location

K2 Medical Research

Maitland, Florida, 32751, United States

Location

Alzheimer's Treatment and Research Center

Stuart, Florida, 34997, United States

Location

Alzheimer's Treatment and Research Center

Wellington, Florida, 33414, United States

Location

Columbus Memory Center, LLC

Columbus, Georgia, 31909, United States

Location

Re:Cognition Health

Fairfax, Virginia, 22031, United States

Location

MeSH Terms

Conditions

Alzheimer Disease

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Limitations and Caveats

The study was terminated due to a strategic decision by the sponsor. No participants received treatment, and no data were collected or evaluated for this study.

Results Point of Contact

• Title: Asceneuron Clinical Research
• Organization: Asceneuron S.A.

clinicaltrials.gov@asceneuron.com

+41213538245

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2024
• First Posted: November 6, 2024
• Study Start: October 16, 2024
• Primary Completion: November 8, 2024
• Study Completion: November 8, 2024
• Last Updated: June 29, 2025
• Results First Posted: June 29, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)