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Investigator Initiated Study for the Safety and Efficacy in Frontotemporal Dementia

ConditionDementia Frontotemporal
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age55-80
SponsorHee-Jin Kim

About this trial

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)

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

Participants with other degenerative brain diseases as determined by the investigator

Participants with other neurological disorders and uncontrolled acute disease

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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

Treatment groups

15 Participants
are divided into 2 treatment groups
Group A: NS101 Treatment GroupActive comparator 1 intervention
Group B: NS101 Placebo GroupPlacebo comparator 1 intervention

Trial outcomes

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.

Time frame
Every Visit for 12month(48weeks)

Secondary outcomes

1

Plasma FAM19A5 Concentration

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

Time frame
Every Visit for 12month(48weeks)
2

Minimum Plasma Concentration of NS101 (Cmin, Ctrough)

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

Time frame
Every Visit for 12month(48weeks)
3

Anti-Drug Antibodies (ADA)

To evaluate immunogenicity following repeated administration of NS101 compared to placebo

Time frame
Every Visit for 12month(48weeks
4

Neutralizing Antibodies (NAbs)

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

Time frame
Every Visit for 12month(48weeks)

Other outcomes

1

Change From Baseline in Neuropsychological Test Scores (FAB)

Neuropsychological assessment \- Score on Frontal Assessment Battery (FAB)

Time frame
Changes from baseline at each assessment time point - Screening, Week 24, Week 36, Week48
2

Change from Baseline in Biomarkers

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

Time frame
Changes from baseline at each assessment time point • Screening, Week 24, Week 36, Week48
3

Change from Baseline in Brain MRI

Neuroimaging \- Brain MRI Measures With Functional MRI (fMRI)

Time frame
Changes from baseline at each assessment time point • Screening, Week 24, Week 36, Week48
4

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

Neuropsychological assessment \- Score on Boston Naming Test (BNT) or Western Aphasia Battery (WAB)

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

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Hee-Jin Kim

Lead sponsor

Hanyang University Seoul Hospital

Sponsor institution

Asan Medical Center

Collaborator

Gangnam Severance Hospital

Collaborator

Konkuk University Medical Center

Collaborator

Source ClinicalTrials.gov

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