Investigator Initiated Study for the Safety and Efficacy in Frontotemporal Dementia

NCT07154485 | Not Yet Recruiting Phase 2

• 1 other identifier: NS101_I2a_FTD
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

Brief Summary

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)

Conditions

Dementia Frontotemporal

Keywords

Frontotemporal Dementia; semantic variant; FTD; sv FTD; PPA; sv PPA

Outcome Measures

Primary Outcomes (1)

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

  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.

  Every Visit for 12month(48weeks)

Secondary Outcomes (4)

• Plasma FAM19A5 Concentration

  Every Visit for 12month(48weeks)

• Minimum Plasma Concentration of NS101 (Cmin, Ctrough)

  Every Visit for 12month(48weeks)

• Anti-Drug Antibodies (ADA)

  Every Visit for 12month(48weeks

• Neutralizing Antibodies (NAbs)

  Every Visit for 12month(48weeks)

Other Outcomes (7)

• Change From Baseline in Neuropsychological Test Scores (FAB)

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

• Change from Baseline in Biomarkers

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

• Change from Baseline in Brain MRI

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

Study Arms (2)

NS101 Treatment Group

ACTIVE COMPARATOR

Participants will receive a two loading dose of NS101 15 mg/kg IV infusion, followed by biweekly dosing for 6 months during the double-blind phase. Those who complete the double-blind phase will be eligible to enter a 3-month open-label extension to receive active treatment.

Drug: NS101 IV infusion

NS101 Placebo Group

PLACEBO COMPARATOR

Participants will receive a two loading dose of NS101 15 mg/kg IV infusion, followed by biweekly dosing for 6 months during the double-blind phase. Those who complete the double-blind phase will be eligible to enter a 3-month open-label extension to receive active treatment.

Drug: Placebo IV Infusion

Interventions

NS101 IV infusion DRUG

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

NS101 Treatment Group

Placebo IV Infusion DRUG

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

NS101 Placebo Group

Eligibility Criteria

• Age: 55 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Hee-Jin Kim: lead
• Asan Medical Center: collaborator
• Gangnam Severance Hospital: collaborator
• Konkuk University Medical Center: collaborator

MeSH Terms

Conditions

Pick Disease of the Brain; Frontotemporal Dementia

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor, Department of Neurology, College of Medicine, Hanyang university

Study Record Dates

• First Submitted: August 12, 2025
• First Posted: September 4, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): August 1, 2028
• Last Updated: September 4, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share