Safety, Tolerability and Efficacy for CGF166 in Patients With Unilateral or Bilateral Severe-to-profound Hearing Loss
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ClinicalTrials.gov Identifier: NCT02132130 |
Recruitment Status :
Completed
First Posted : May 7, 2014
Last Update Posted : July 15, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Unilateral Severe to Profound Hearing Loss OR Bilateral Severe to Profound Hearing Loss | Drug: CGF166 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 22 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Three-part, Multicenter, Open Label, Single Dose Study to Assess the Safety, Tolerability, and Efficacy of Intra Labyrinthine (IL) CGF166 in Patients With Severe-to-profound Hearing Loss |
Actual Study Start Date : | June 23, 2014 |
Actual Primary Completion Date : | December 9, 2019 |
Actual Study Completion Date : | December 9, 2019 |

Arm | Intervention/treatment |
---|---|
Experimental: CGF166 dose 20 uL
single dose volume #1
|
Drug: CGF166
CGF166 is a recombinant adenovirus 5 (Ad5) vector containing the human Atonal transcription factor (Hath1) cDNA for administration via intra-labyrinthine infusion |
Experimental: CGF166 dose 30 and 40 uL
single dose volume #2
|
Drug: CGF166
CGF166 is a recombinant adenovirus 5 (Ad5) vector containing the human Atonal transcription factor (Hath1) cDNA for administration via intra-labyrinthine infusion |
Experimental: CGF166 dose 40 uL
single dose volume #3
|
Drug: CGF166
CGF166 is a recombinant adenovirus 5 (Ad5) vector containing the human Atonal transcription factor (Hath1) cDNA for administration via intra-labyrinthine infusion |
Experimental: CGF166 dose 60 uL
single dose volume #4
|
Drug: CGF166
CGF166 is a recombinant adenovirus 5 (Ad5) vector containing the human Atonal transcription factor (Hath1) cDNA for administration via intra-labyrinthine infusion |
Experimental: CFG166 dose 30 uL
Single dose volume #5
|
Drug: CGF166
CGF166 is a recombinant adenovirus 5 (Ad5) vector containing the human Atonal transcription factor (Hath1) cDNA for administration via intra-labyrinthine infusion |
- Number of patients reported adverse events [ Time Frame: 24 months ]Number of patients reported with total adverse events, serious adverse events and death as an assessment of safety and tolerability
- Change in pure tone audiometry compared to pretreatment values [ Time Frame: 24 months ]Sensory thresholds at pure tone frequencies from 0.125 KHz through 16 KHz will be measured using standard audiometric techniques.
- Change in brainstem auditory evoked responses (BAER) compared to pretreatment values [ Time Frame: 24 months ]BAERs will be assessed with standard techniques for clinically significant threshold improvements compared to baseline levels.
- effects of CGF166 on various assessments of vestibular function compared to pretreatment values [ Time Frame: 24 months ]Assessments of the vestibular organs for clinically relevant functional improvement
- Changes in auditory functions (speech recognition) and vestibular functions before and after IL infusion of CGF166 between the study ear and the contralateral ear [ Time Frame: 24 months ]Clinically signficant speech recognition improvement (word and/or sentence) following treatment

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
For all Parts A, B and C of the study,
Inclusion criteria:
- Written informed consent must be obtained before any assessment is performed.
-
For all Parts A, B and C of the study, male or female patients, 18 to 75 years old, inclusive, with severe-to-profound bilateral hearing loss or unilateral hearing loss with intact vestibular function in the nonoperative ear. Non-fluctuating severe-to-profound hearing loss is required for the study ear and is defined as:
- PTA within 10 dB of the PTA obtained at least 11 months previously.
- Word recognition within 20% of previous test at least 11 months previously
- Candidate ear ("study ear"): Minimal residual hearing based on the pure tone average of 0.5, 1, 2, and 4 kHz thresholds of ≤110 dB HL
- Candidate ear ("study ear"): Pure tone audiometric thresholds of ≥50 dB HL for each testable octave frequency of 0.125 and 0.250 kHz, ≥70 dB HL for each testable octave frequency from 0.5 through 8 kHz and sentence recognition scores ≤50% at screening.
6. Patients with intact vestibular function in at least one ear (non-study ear) as measured by vestibular evoked myogenic potential (VEMP) 7. Able to communicate well with the investigator, to understand and comply with the requirements of the study 8. MRI scan within 6 months or at screening to confirm suitability for inner ear surgery 9. Patients must weigh at least 40 kg to participate in the study, and must have a body mass index (BMI) <45 kg/m2. BMI = Body weight (kg) / [Height (m)]2
Exclusion Criteria:
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02132130
United States, Kansas | |
Novartis Investigative Site | |
Kansas City, Kansas, United States, 66160 | |
United States, Maryland | |
Novartis Investigative Site | |
Baltimore, Maryland, United States, 21287 | |
United States, New York | |
Novartis Investigative Site | |
New York, New York, United States, 10032 | |
United States, Oregon | |
Novartis Investigative Site | |
Portland, Oregon, United States, 97239 |
Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
Responsible Party: | Novartis Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT02132130 |
Other Study ID Numbers: |
CCGF166X2201 |
First Posted: | May 7, 2014 Key Record Dates |
Last Update Posted: | July 15, 2020 |
Last Verified: | July 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Plan Description: | Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
gene therapy unilateral hearing loss bilateral hearing loss balance |
vestibular cochlear implant hearing restoration |
Hearing Loss Deafness Hearing Disorders Ear Diseases |
Otorhinolaryngologic Diseases Sensation Disorders Neurologic Manifestations Nervous System Diseases |