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NCT Number: NCT07154485

Investigator Initiated Study for the Safety and Efficacy in Frontotemporal Dementia

The purpose of this clinical trial is to evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), immunogenicity, and efficacy of multiple intravenous administrations of the investigational drug NS101, compared to placebo, in patients with semantic variant primary progressive aphasia (svPPA), a subtype of frontotemporal dementia (FTD)

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Key information

Conditions

Dementia Frontotemporal Brain Diseases Central Nervous System Diseases Dementia Frontotemporal Dementia Frontotemporal Lobar Degeneration Mental Disorders Metabolic Diseases Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Nutritional and Metabolic Diseases Pick Disease of the Brain Proteostasis Deficiencies TDP-43 Proteinopathies

Age range

55 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Participants diagnosed at screening with mild to moderate frontotemporal dementia, semantic variant (svFTD), defined by a CDR® Plus NACC FTLD Sum of Boxes (SOB) score between 4.5 and 15.5.
  • Participants confirmed to be amyloid-negative based on Amyloid PET scan or CSF results within the past 36 months.

Key Exclusion Criteria:

  • Participants with other degenerative brain diseases as determined by the investigator
  • Participants with other neurological disorders and uncontrolled acute disease

Treatment and study plan

NS101 IV infusion

Drug

NS101 is anti FAM19A5 antibody expected to play as a synapse organizer and reversing synapse dysfunction in various neurological diseases

Placebo IV Infusion

Drug

Placebo (i.e. fake drug without active pharmaceutical ingredient) of NS101

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)

    Time frame: Every Visit for 12month(48weeks)

    To evaluate the safety and tolerability of repeated administration of NS101 compared to placebo in patients with semantic variant primary progressive aphasia (svPPA), a subtype of frontotemporal dementia (FTD).

    <primary efficacy endpoint> The incidence, causality, and severity of adverse events (AEs), serious adverse events (SAEs), adverse drug reactions (ADRs), and serious adverse drug reactions (SADRs) by group.

Secondary outcomes

  1. Plasma FAM19A5 Concentration

    Time frame: Every Visit for 12month(48weeks)

    To assess the pharmacodynamic (PD) effect of NS101 compared to placebo in patients with svPPA

  2. Minimum Plasma Concentration of NS101 (Cmin, Ctrough)

    Time frame: Every Visit for 12month(48weeks)

    To evaluate the pharmacokinetics (PK) of repeated administration of NS101 compared to placebo in patients with svPPA

  3. Anti-Drug Antibodies (ADA)

    Time frame: Every Visit for 12month(48weeks

    To evaluate immunogenicity following repeated administration of NS101 compared to placebo

  4. Neutralizing Antibodies (NAbs)

    Time frame: Every Visit for 12month(48weeks)

    To evaluate the presence of neutralizing antibodies following repeated administration of NS101 compared to placebo

Other outcomes

  1. Change From Baseline in Neuropsychological Test Scores (FAB)

    Time frame: Changes from baseline at each assessment time point - Screening, Week 24, Week 36, Week48

    Neuropsychological assessment

    • Score on Frontal Assessment Battery (FAB)

  2. Change from Baseline in Biomarkers

    Time frame: Changes from baseline at each assessment time point • Screening, Week 24, Week 36, Week48

    Biomarkers

    • Concentration of Neurofilament Light Chain (NfL) [pg/mL]
    • Concentration of Glial Fibrillary Acidic Protein (GFAP) [pg/mL]

  3. Change from Baseline in Brain MRI

    Time frame: Changes from baseline at each assessment time point • Screening, Week 24, Week 36, Week48

    Neuroimaging

    • Brain MRI Measures With Functional MRI (fMRI)

  4. Change From Baseline in Neuropsychological Test Scores (BNT/WAB)

    Time frame: Changes from baseline at each assessment time point - Screening, Week 24, Week 36, Week48

    Neuropsychological assessment

    • Score on Boston Naming Test (BNT) or Western Aphasia Battery (WAB)

  5. Change From Baseline in Neuropsychological Test Scores (ADCS-ADL)

    Time frame: Changes from baseline at each assessment time point - Screening, Week 24, Week 36, Week48

    Neuropsychological assessment

    • Score on Alzheimer's Disease Cooperative Study-Activities of Daily Living (ADCS-ADL)

  6. Change From Baseline in Neuropsychological Test Scores (FBI)

    Time frame: Changes from baseline at each assessment time point - Screening, Week 24, Week 36, Week48

    Neuropsychological assessment

    • Score on Frontal Behavioral Inventory (FBI)

  7. Change From Baseline in Neuropsychological Test Scores (C-SSRS)

    Time frame: Changes from baseline at each assessment time point - Screening, Week 24, Week 36, Week48

    Neuropsychological assessment

    • Score on Columbia-Suicide Severity Rating Scale (C-SSRS)

Sponsors and collaborators

Lead sponsor

Hee-Jin Kim

Other

Collaborators

  • Asan Medical Center
  • Gangnam Severance Hospital
  • Konkuk University Medical Center

Registry information

Official study title

A Prospective, Multicenter, Randomized, Double-blind, Placebo-controlled, Investigator-initiated Phase 2a Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics/Pharmacodynamics (PK/PD), and Exploratory Efficacy of Multiple Doses of NS101 in Patients With Semantic Variant Primary Progressive Aphasia (svPPA), a Subtype of Frontotemporal Dementia (FTD)

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Sep 4, 2025
Registry last updated
Sep 4, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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