A Phase 1b/2a, Study Evaluating the Safety, PK/PD and Efficacy of NS101 in Healthy Volunteers and SSNHL Patients

A Phase 1b/2a, Double-blinded, Placebo-controlled, Multiple Doses, 2 Step-up Study Evaluating the Safety, Tolerability, PK/PD and Efficacy of Systemic NS101 in Healthy Volunteers and SSNHL Patients

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06249919

Enrollment

138

Registered

2024-02-08

Start date

2024-01-19

Completion date

2027-01-31

Last updated

2025-08-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Seneorineural Deafness, Sudden Sensorineural Hearing Loss

Keywords

Sensorineural disease, sudden sensorineural hearing loss, sensorineural hearing loss, sudden deafness, cochlear synaptopathy, hidden auditory synaptopathy

Brief summary

The goal of this 2 step-up, exploratory study is to test safey, tolerability and PK/PD profiles in healthy volunteers and safety, tolerability and efficacy in sudden sensorineural hearing loss patients as an early salvage therapy. The main questions it aims to answer are: 1. whether is it safe and tolerable when healthy volunteers and sudden sensorineural hearing loss patients take multiple doses of NS101 against FAM19A5 2. whether is it effective in reversing hearing capability in sudden sensorineural hearing loss patients who fails to show sufficient recovery despite of oral standard steroid therapy. Patients and heathly volunteers will be given NS101 15mg/kg or 30mg/kg systemically less than 3 months per protocol. This is placebo controlled, double blinded study, which means there will be a group who receives placebo (i.e. fake drug) for study purpose.

Interventions

BIOLOGICALNS101

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

BIOLOGICALPlacebo

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

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Investigator, Outcomes Assessor)

Intervention model description

A Phase 1b/2a, prospective, randomized, double-blind, placebo-controlled, multicenter, dose-escalation, exploratory study evaluating the safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), and efficacy of multiple doses of NS101 in healthy adults and patients with sudden Sensorineural Hearing Loss

Eligibility

Sex/Gender

ALL

Age

19 Years to 70 Years

Healthy volunteers

Yes

Inclusion criteria

Part A: healthy volunteers Part B: Inclusion Criteria: * unilateral ideopathic SSNHL greater than 45dB at the average of 4 frequencies in PTA (contralateral hearing is less than 30dB) * Subject who has completed investigator-recognized standard treatment within 30 days of symptom onset and is able to undergo randomization visit within 35 days. * Subjects who, in the opinion of the investigator, have received sufficient systemic steroid therapy for approximately 2 weeks recovery * A combination regimen consisting of approximately 2 weeks of systemic steroid therapy and intratympanic steroid administration

Exclusion criteria

* other otologic or systemic diseases * retrocochlear lesion

Design outcomes

Primary

Measure	Time frame	Description
Phase 1b part: Change from Baseline in Safety Profiles	Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43	Adverse Event, Serious Adverse Event, ratio of Treatment related Serisous Adverse Event with causality & severity will be descriptively measured and collected in the following time frames
Phase 2a part: Changes from Baseline in Safety Profiles	Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20	Adverse Event, Serious Adverse Event, ratio of Treatment related Serisous Adverse Event with causality & severity will be descriptively measured and collected in the following time frames

Secondary

Measure	Time frame	Description
Phase 1b part: Change from Baseline in PK/PD profiles & Immunological Assay	Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43	Concentrations, Areas Under the Curve versus time curve, Times and Volumes of Distribution of various status verus time curve
Phase 2a part : Change from Baseline in PK/PD profiles & immunological Assay	Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20	Concentrations, Areas Under the Curve versus time curve, Times and Volumes of Distribution of various status verus time curve

Other

Measure	Time frame	Description
Phase 2a part : Change from Baseline in Pure Tone Audiometry, Speech Discrimination Scores and Tinnitus Handicap Inventory in Sudden Sensorineural Hearing Loss Patients	Baseline, Week4, Week8, Week12, Week16, Week20	hearing capacity of thresholds (e.g. PTA, ABR, DPOAE, ECochG), speech discrimination (e.g. SRT, SDS) and tinnitus (e.g. THI, VAS)

Countries

South Korea

Contacts

Primary ContactYunkyung Choi

clinical@neuracles.com+82269490409

Backup ContactSuhyun Cho