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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
Results First Posted : March 30, 2021
Last Update Posted : March 30, 2021
Sponsor:
Information provided by (Responsible Party):
Novartis ( 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).

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

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.

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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

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: week 52 ]
    AE tables are below in the Adverse Events section of this report.

  2. Number of Adverse Events [ Time Frame: week 52 ]
    AE tables are below in the Adverse Events section of this report.

  3. Summary of Pure Tone Audiometry in Treated Ear Compared to Pretreatment Values [ Time Frame: Days 29, 57, 85, 113, 141, 169, 358, 537, 600 ]
    Summary of pure tone audiometry air conduction thresholds at frequency 0.125 KHz

  4. Summary Pure Tone Audiometry Bone Conduction Thresholds in Treated Ear by Time and Frequency [ Time Frame: 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

  5. Summary of Change From Baseline in Treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit [ Time Frame: 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.

  6. Summary of Change From Baseline in Non-treated Ear's Pure Tone Audiometry Air Conduction Threshold by Frequency for Last Study Visit [ Time Frame: 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.


Secondary Outcome Measures :
  1. Number of Participants With Change in Brainstem Auditory Evoked Responses (BAER) Compared to Pretreatment Values [ Time Frame: 24 months ]
    BAERs was assessed with standard techniques for clinically significant threshold improvements compared to baseline levels.

  2. Number of Participants With Response in Vestibular Function in Treated Ear Compared to Pretreatment Values [ Time Frame: 24 months ]
    Response in vestibular assessments (Head impulse test (HIT), Vestibular evoked myogenic potential (VEMP), Subjective visual vertical (SVV)) to CGF166.

  3. 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 [ Time Frame: 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.



Information from the National Library of Medicine

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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
Criteria

For all Parts A, B and C of the study,

Inclusion criteria:

  1. Written informed consent must be obtained before any assessment is performed.
  2. 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
  3. 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
  4. 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:

  1. Patients with hearing loss caused by genetic/developmental disorders, e.g., cochlea aplasia
  2. 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;
  3. Patients with a history of cochlear implant in the study ear
  4. 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
  5. Patients who will require ototoxic drugs as routine therapy over the course of the study, for example cystic fibrosis patients
  6. Any contraindication to the planned surgery or anesthesia as determined by the surgeon or anesthesiologist
  7. Previous surgery in the study ear
  8. 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
  9. Pregnant women
  10. Abnormal vital signs and/or ECG that suggest potential contraindication for planned study anesthesia
  11. Past serious adverse reaction to anesthesia
  12. Meniere's Disease
  13. History of radiation therapy to the head and neck
  14. Participation in a clinical trial within the last 30 days
  15. Immunocompromised patients, as judged by the investigators based on patient history, physical exam and CBC

Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Novartis ( Novartis Pharmaceuticals ):
Study Protocol  [PDF] April 30, 2019
Statistical Analysis Plan  [PDF] August 17, 2018

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02132130    
Other Study ID Numbers: CCGF166X2201
First Posted: May 7, 2014    Key Record Dates
Results First Posted: March 30, 2021
Last Update Posted: March 30, 2021
Last Verified: March 2021
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


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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
gene therapy
unilateral hearing loss
bilateral hearing loss
balance
vestibular
cochlear implant
hearing restoration
Additional relevant MeSH terms:
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Hearing Loss
Deafness
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases