Investigator Initiated Study for the Safety and Efficacy in Frontotemporal Dementia

Not Applicable

Not yet recruiting

• Conditions: Dementia Frontotemporal
• Interventions: Drug: NS101 IV infusion; Drug: Placebo IV Infusion

• Registration Number: NCT07154485
• Lead Sponsor: Hee-Jin Kim

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)

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-08
• Study First Submitted: 2025-08-12
• Study First Submitted QC: 2025-09-03
• Last Update Submitted: 2025-09-03
• Study First Posted: 2025-09-04
• Last Update Posted: 2025-09-04
• Study Start: 2025-10-01
• Primary Completion: 2027-08-01
• Study Completion: 2028-08-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
NS101 Treatment Group	NS101 IV infusion	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.
NS101 Placebo Group	Placebo IV Infusion	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)	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 Outcome Measures

Name	Time	Method
Plasma FAM19A5 Concentration	Every Visit for 12month(48weeks)	To assess the pharmacodynamic (PD) effect of NS101 compared to placebo in patients with svPPA
Minimum Plasma Concentration of NS101 (Cmin, Ctrough)	Every Visit for 12month(48weeks)	To evaluate the pharmacokinetics (PK) of repeated administration of NS101 compared to placebo in patients with svPPA
Anti-Drug Antibodies (ADA)	Every Visit for 12month(48weeks	To evaluate immunogenicity following repeated administration of NS101 compared to placebo
Neutralizing Antibodies (NAbs)	Every Visit for 12month(48weeks)	To evaluate the presence of neutralizing antibodies following repeated administration of NS101 compared to placebo