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

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.

Study Overview

• Status: Recruiting
• Conditions: Sudden Sensorineural Hearing Loss; Seneorineural Deafness
• Intervention / Treatment: Biological: NS101; Biological: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 138
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Yunkyung Choi; Phone Number: +82269490409; Email: clinical@neuracles.com
• Study Contact Backup: Name: Suhyun Cho; Phone Number: +82269490409; Email: clinical@neuracles.com

Study Locations

• Korea, Republic of
  • Bucheon, Korea, Republic of
    • Not yet recruiting
    • SCH University Hospital Bucheon
    • Contact: JD Lee, MD/PhD
  • Gwangju, Korea, Republic of
    • Recruiting
    • Chonam National University Hospital
    • Contact: HH Cho, MD/PhD
  • Ilsan, Korea, Republic of
    • Recruiting
    • Inje University Ilsan Paik Hospital
    • Contact: JM Lee, MD/PhD
  • Ilsan, Korea, Republic of
    • Not yet recruiting
    • MyongJi Hospital
    • Contact: DB Sim, MD/PhD
  • Pusan, Korea, Republic of
    • Recruiting
    • Pusan National University Hospital
    • Contact: SG Kong, MD/PhD
  • Pyeongchon, Korea, Republic of
    • Recruiting
    • Hallym Sacred Heart Hospital
    • Contact: HJ Lee, MD/PhD
  • Seongnam, Korea, Republic of
    • Recruiting
    • Seoul National University Bundang Hospital
    • Contact: JJ Song, MD/PhD
  • Seoul, Korea, Republic of
    • Recruiting
    • Asan Medical Center
    • Contact: HJ Park, MD/PhD
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul National University Hospital
    • Contact: MH Seo, MD/PhD
  • Seoul, Korea, Republic of
    • Recruiting
    • Samsung Medical Center
    • Contact: IJ Moon, MD/PhD
  • Seoul, Korea, Republic of
    • Recruiting
    • Korea University Anam Hospital
    • Contact: JY Park, MD/PhD
  • Seoul, Korea, Republic of
    • Recruiting
    • Yonsei University Severance Hospital
    • Contact: JY Choi, MD/PhD
  • Seoul, Korea, Republic of
    • Not yet recruiting
    • Korea University Anam Hospital
    • Contact: GJ Im, MD/PhD
  • Seoul, Korea, Republic of
    • Recruiting
    • Catholic University Seoul St. Mary's Hospital
    • Contact: SN Park, MD/PhD
  • Suwon, Korea, Republic of
    • Not yet recruiting
    • Ajou University Hospital
    • Contact: YH Jeong, MD/PhD
  • Wonju, Korea, Republic of
    • Recruiting
    • Wonju Severance Christian Hospital
    • Contact: YJ Seo, MD/PhD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Group A; NS101 15mg/kg IV infusion Biweekly for 6weeks in Healthy Volunteers (active:placebo=6:3)	Biological: NS101; NS101 is anti FAM19A5 antibody expected to play as a synapse organizer and reversing synapse dysfunction in various neurological diseases; Biological: Placebo; Placebo (i.e. fake drug without active pharmaceutical ingredient) of NS101
Experimental: Group B; NS101 30mg/kg IV infusion Biweekly for 6weeks in Healthy Volunteers (active:placebo=6:3)	Biological: NS101; NS101 is anti FAM19A5 antibody expected to play as a synapse organizer and reversing synapse dysfunction in various neurological diseases; Biological: Placebo; Placebo (i.e. fake drug without active pharmaceutical ingredient) of NS101
Active Comparator: Cohort A; NS101 15mg/kg IV infusion Biweekly for 12weeks in Sudden Sensorineural Hearing Loss patients	Biological: NS101; NS101 is anti FAM19A5 antibody expected to play as a synapse organizer and reversing synapse dysfunction in various neurological diseases
Placebo Comparator: Cohort B; Placebo 15mg/kg IV infusion Biweekly for 12weeks in Sudden Sensorineural Hearing Loss patients	Biological: Placebo; Placebo (i.e. fake drug without active pharmaceutical ingredient) of NS101

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase 1b part: Change from Baseline in Safety Profiles	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	Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43
Phase 2a part: Changes from Baseline in Safety Profiles	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	Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20

Secondary Outcome Measures

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

Other Outcome Measures

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

Collaborators and Investigators

• Sponsor: Neuracle Science Co., LTD.
• Collaborators: Samsung Medical Center; Korea University Anam Hospital; Severance Hospital
• Investigators: Study Director: Joseph Park, M.D., Neuracle Science

Study record dates

Study Major Dates

• Study Start (Actual): January 19, 2024
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): January 31, 2027

Study Registration Dates

• First Submitted: January 12, 2024
• First Submitted That Met QC Criteria: January 31, 2024
• First Posted (Actual): February 8, 2024

Study Record Updates

• Last Update Posted (Actual): August 13, 2025
• Last Update Submitted That Met QC Criteria: August 7, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: sensorineural hearing loss; sudden sensorineural hearing loss; sudden deafness; Sensorineural disease; cochlear synaptopathy; hidden auditory synaptopathy
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Ear Diseases; Hearing Disorders; Hearing Loss; Deafness; Hearing Loss, Sensorineural
• Other Study ID Numbers: NS101_P2_04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Currently there is no plan to share IPD at this stage

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No