Clinical trial NCT06249919: A Phase 1b/2a, Study Evaluating the Safety, PK/PD and Efficacy of NS101 in Healthy Volunteers and SSNHL Patients
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Seneorineural Deafness
Sudden Sensorineural Hearing Loss
Keywords: Sensorineural disease
sudden sensorineural hearing loss
sensorineural hearing loss
sudden deafness
cochlear synaptopathy
hidden auditory synaptopathy
Lead sponsor: Neuracle Science Co., LTD.
Sponsor class: INDUSTRY
Collaborators: name: Samsung Medical Center
name: Severance Hospital
name: Korea University Anam Hospital
First posted: 2024-02-08
Enrollment: 118
Sex: ALL
Minimum age (years): 19
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
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
Primary purpose: TREATMENT

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.


Eligibility criteria: Part A: healthy volunteers

Part B:

Inclusion Criteria:

* unilateral ideopathic SSNHL greater than 55dB at the average of 4 frequencies in PTA (contralateral hearing is less than 30dB)
* those who started standard treatment within 5 days after symptom onset
* those who were treated with oral steroids for 14 days, but incomplete recovery was confirmed as type III/IV according to Siegel's criteria at the end of the 14-day treatment

Exclusion Criteria:

* other otologic or systemic diseases
* retrocochlear lesion

Primary outcomes:
measure: Phase 1b part: Change from Baseline in Safety Profiles
timeFrame: Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43
measure: Phase 2a part: Changes from Baseline in Safety Profiles
timeFrame: Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20
Secondary outcomes:
measure: Phase 1b part: Change from Baseline in PK/PD profiles & Immunological Assay
timeFrame: Baseline, Day1, Day2, Day3, Day14, Day15, Day16, Day17, Day28, Day29, Day30, Day32, Day43
measure: Phase 2a part : Change from Baseline in PK/PD profiles & immunological Assay
timeFrame: Baseline, Week1, Week2, Week4, Week6, Week8, Week10, Week12, Week16, Week20
Other outcomes:
measure: Phase 2a part : Change from Baseline in Pure Tone Audiometry, Speech Discrimination Scores and Tinnitus Handicap Inventory in Sudden Sensorineural Hearing Loss Patients
timeFrame: Baseline, Week4, Week8, Week12, Week16, Week20
Locations:
city: Bucheon
country: Korea, Republic of
facility: SCH University Hospital Bucheon
city: Gwangju
country: Korea, Republic of
facility: Chonam National University Hospital
city: Ilsan
country: Korea, Republic of
facility: Inje University Ilsan Paik Hospital
city: Ilsan
country: Korea, Republic of
facility: Myongji Hospital
city: Pusan
country: Korea, Republic of
facility: Pusan National University Hospital
city: Pyeongchon
country: Korea, Republic of
facility: Hallym Sacred Heart Hospital
city: Seongnam
country: Korea, Republic of
facility: Seoul National University Bundang Hospital
city: Seoul
country: Korea, Republic of
facility: Asan Medical Center
city: Seoul
country: Korea, Republic of
facility: Catholic University Seoul St. Mary's Hospital
city: Seoul
country: Korea, Republic of
facility: Korea University Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Korea University Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Yonsei University Severance Hospital
city: Suwon
country: Korea, Republic of
facility: Ajou University Hospital
city: Wonju
country: Korea, Republic of
facility: Wonju Severance Christian Hospital
Study officials:
affiliation: Neuracle Science
name: Joseph Park, M.D.
role: STUDY_DIRECTOR
References: