Safety, Tolerability and Efficacy for CGF166 in Patients With Unilateral or Bilateral Severe-to-profound Hearing Loss
- Conditions
- Unilateral Severe to Profound Hearing Loss OR Bilateral Severe to Profound Hearing Loss
- Interventions
- Registration Number
- NCT02132130
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
The goal of the study was to evaluate the safety, tolerability, and the potential ability of CGF166 delivered through IL-infusion to improve hearing. CGF166 is a recombinant adenovirus 5 (Ad5) vector containing a cDNA encoding the human Atonal transcription factor (Hath1).
- Detailed Description
This study evaluated the safety, tolerability, and potential efficacy of CGF166 and the associated delivery procedures in patients with severe-to-profound unilateral or bilateral hearing loss. Eligible patients were required to have documented, non-fluctuating hearing loss.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 22
Not provided
- Patients with hearing loss caused by genetic/developmental disorders, e.g., cochlea aplasia
- Patients with existing conductive hearing loss or mixed hearing loss as judged by the Principal Investigator following a thorough review of all of the trial hearing assessments;
- Patients with a history of cochlear implant in the study ear
- Hearing loss due to any other cause that would not be expected to respond to hair cell regeneration, for example mechanical trauma or central auditory lesions or lack of an auditory nerve
- Patients who will require ototoxic drugs as routine therapy over the course of the study, for example cystic fibrosis patients
- Any contraindication to the planned surgery or anesthesia as determined by the surgeon or anesthesiologist
- Previous surgery in the study ear
- Any otological history, such as chronic otitis, cholesteatoma, tympanic membrane perforation, that suggests poor candidacy for cochlear implant or inner ear surgery or suggests potential interference with study auditory or vestibular function tests
- Pregnant women
- Abnormal vital signs and/or ECG that suggest potential contraindication for planned study anesthesia
- Past serious adverse reaction to anesthesia
- Meniere's Disease
- History of radiation therapy to the head and neck
- Participation in a clinical trial within the last 30 days
- Immunocompromised patients, as judged by the investigators based on patient history, physical exam and CBC
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description CGF166 dose 40 uL CGF166 single dose volume #3 CGF166 dose 20 uL CGF166 single dose volume #1 CGF166 dose 30 and 40 uL CGF166 single dose volume #2 CGF166 dose 60 uL CGF166 single dose volume #4 CFG166 dose 30 uL CGF166 Single dose volume #5
- Primary Outcome Measures
Name Time Method Summary of Change From Baseline in Treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit Week 52 Summary of change from baseline in pure tone audiometry air conduction threshold by frequency for last study visit is presented in table below.
Summary of Change From Baseline in Non-treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit Week 52 Summary of change from baseline in Non-treated ear's pure tone audiometry air conduction threshold by frequency for last study visit is presented in table below.
Number of Participants With Adverse Events week 52 AE tables are below in the Adverse Events section of this report.
Number of Adverse Events week 52 AE tables are below in the Adverse Events section of this report.
Summary of Pure Tone Audiometry in Treated Ear Compared to Pretreatment Values Days 29, 57, 85, 113, 141, 169, 358, 537, 600 Summary of pure tone audiometry air conduction thresholds at frequency 0.125 KHz
Summary Pure Tone Audiometry Bone Conduction Thresholds in Treated Ear by Time and Frequency Days 29, 57, 85, 113, 141, 169, 358,537, EoS Summary of pure tone audiometry bone conduction thresholds by time and frequency 0.250 KHz
- Secondary Outcome Measures
Name Time Method Number of Participants With Response in Vestibular Function in Treated Ear Compared to Pretreatment Values 24 months Response in vestibular assessments (Head impulse test (HIT), Vestibular evoked myogenic potential (VEMP), Subjective visual vertical (SVV)) to CGF166.
Number of Participants With Changes in Auditory Functions (Speech Recognition) and Vestibular Functions Before and After IL Infusion of CGF166 Between the Study Ear and the Contralateral Ear 24 months Clinically signficant speech recognition improvement (word and/or sentence) following treatment. The individual auditory assessments were speech audiometry, AzBio sentence test, consonant nucleus consonant test, word recognition, Hearing-in-Noise Test (HINT), Brainstem auditory evoked response evaluations (BAER), Distortion product otoacoustic emission testing (DPOE) and shoebox audiometry.
Number of Participants With Change in Brainstem Auditory Evoked Responses (BAER) Compared to Pretreatment Values 24 months BAERs was assessed with standard techniques for clinically significant threshold improvements compared to baseline levels.
Trial Locations
- Locations (1)
Novartis Investigative Site
🇺🇸Portland, Oregon, United States