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History of Changes for Study: NCT02132130
Safety, Tolerability and Efficacy for CGF166 in Patients With Bilateral Severe-to-profound Hearing Loss
Latest version (submitted October 7, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 May 6, 2014 None (earliest Version on record)
2 May 20, 2014 Study Status, Conditions, Eligibility, Outcome Measures and Arms and Interventions
3 June 2, 2014 Recruitment Status, Study Status and Contacts/Locations
4 November 19, 2014 Contacts/Locations, Study Status and Eligibility
5 February 12, 2015 Study Status, Contacts/Locations, Eligibility and Oversight
6 May 28, 2015 Study Status and Contacts/Locations
7 October 13, 2015 Study Status
8 March 7, 2016 Study Status
9 March 8, 2016 Study Description and Study Status
10 May 9, 2016 Study Status and Study Design
11 June 22, 2016 Study Status and Study Design
12 March 14, 2017 Study Status, Oversight and Conditions
13 March 27, 2017 Conditions and Study Status
14 April 3, 2017 Study Status
15 April 20, 2017 Oversight, IPDSharing, Contacts/Locations and Study Status
16 March 26, 2018 Outcome Measures, Arms and Interventions, Contacts/Locations, Conditions, Study Description, Study Status, Eligibility and Study Identification
17 April 3, 2018 Study Status, Contacts/Locations and Eligibility
18 August 20, 2018 Study Status, Contacts/Locations and Study Design
19 October 22, 2018 Study Status
20 November 8, 2018 Contacts/Locations, Study Status, Eligibility and Study Design
21 May 9, 2019 Study Status and Contacts/Locations
22 August 28, 2019 Study Status
23 November 14, 2019 Recruitment Status, Study Status, Contacts/Locations and Study Design
24 March 18, 2020 Recruitment Status and Study Status
25 July 14, 2020 Study Status
Show
Results Submission Events
26 March 3, 2021 Outcome Measures, Study Status, Study Description, Document Section and Results
27 October 7, 2021 Study Status and References
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Study NCT02132130
Submitted Date:  May 6, 2014 (v1)

Open or close this module Study Identification
Unique Protocol ID: CCGF166X2201
Brief Title: Safety, Tolerability and Efficacy for CGF166 in Patients With Bilateral Severe-to-profound Hearing Loss
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
Secondary IDs:
Open or close this module Study Status
Record Verification: May 2014
Overall Status: Not yet recruiting
Study Start: May 2014
Primary Completion: September 2015 [Anticipated]
Study Completion: September 2015 [Anticipated]
First Submitted: May 5, 2014
First Submitted that
Met QC Criteria:
May 6, 2014
First Posted: May 7, 2014 [Estimate]
Last Update Submitted that
Met QC Criteria:
May 6, 2014
Last Update Posted: May 7, 2014 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Novartis Pharmaceuticals
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: No
Open or close this module Study Description
Brief Summary: The goal of the current study is to evaluate the safety, tolerability, and the potential ability of CGF166 delivered through IL-infusion to improve hearing and vestibular function. CGF166 is a recombinant adenovirus 5 (Ad5) vector containing a cDNA encoding the human Atonal transcription factor (Hath1).
Detailed Description: The current study will evaluate the safety, tolerability, and potential efficacy of CGF166 and the associated delivery procedures in patients with severe-to-profound bilateral hearing loss. Eligible patients are required to have documented, non-fluctuating hearing loss. Part A will include a safety and tolerability cohort (N=3). Patient dosing will be staggered; dosing the next patient in a cohort will be based on a safety review of all available data through 4 weeks post-dose of the previously dosed patient(s). Part B includes a volumetric escalation design to evaluate infusion volumes of the same CGF166 concentration (5.0 x 10E11vp/mL) in 4 cohorts of patients (n=3/cohort; total of 12 patients). Part C is an expansion cohort of the highest safe and tolerable dose identified in Part B, for further assessment of efficacy.
Open or close this module Conditions
Conditions: Severe-to-profound Bilateral Hearing Loss
Keywords: gene therapy
hearing loss
balance
vestibular
cochlear implant
intact vestibular function
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms: 6
Masking: None (Open Label)
Allocation: Non-Randomized
Enrollment: 45 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
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 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 60 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 80 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: CGF166 dose 90 uL
Singel 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
Experimental: CFG166 dose TBD uL
Single dose volume #6
Drug: CGF166
CGF166 is a recombinant adenovirus 5 (Ad5) vector containing the human Atonal transcription factor (Hath1) cDNA for administration via intra-labyrinthine infusion
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Number of patients reported with total adverse events, serious adverse events and death as an assessment of safety and tolerability
[ Time Frame: 6 months ]

2. Change in pure tone audiometry compared to pretreatment values
[ Time Frame: 6 months ]

Sensory thresholds at pure tone frequencies from 0.125 KHz through 16 KHz will be measured using standard audiometric techniques.
Secondary Outcome Measures:
1. Change in otoacoustic emission (OAE) testing compared to pretreatment values
[ Time Frame: 6 months ]

Standard OAE assessments will be performed to evaluate for a clinically significant improvement in signal to noise ratio 6 months following treatment.
2. Change in brainstem auditory evoked responses (BAER) compared to pretreatment values
[ Time Frame: 6 months ]

BAERs will be assessed with standard techniques for clinically significant threshold improvements compared to baseline levels.
3. effects of CGF166 on various assessments of vestibular function compared to pretreatment values
[ Time Frame: 6 months ]

Assessments of the vestibular organs for clinically relevant functional improvement
4. 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: 6 months ]

Clinically signfiicant speech recognition improvement (word and/or sentence) following treatment
Open or close this module Eligibility
Minimum Age: 21 Years
Maximum Age: 70 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

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

Inclusion criteria:

  1. Male or female patients, 21 to 70 years old, inclusive, with severe-to-profound bilateral hearing loss with intact vestibular function in the non-operative ear. Non-fluctuating severe-to-profound hearing loss is required for the study ear and is defined as:
  2. Pure-tone average (PTA) within 10 dB of the PTA obtained at least 11 months previously
  3. Word recognition within 20% of previous test at least 11 months previously
  4. Hearing loss has been diagnosed within 10 years of the expected date of treatment
  5. Candidate ear ("study ear") pure tone audiometric thresholds of ≥70 dB HL for each testable octave frequency from 0.125 through 8 kHz and sentence recognition ≤50% at screening
  6. Contralateral ear ("non-study ear"): Despite use of a hearing aid meets cochlear implantation criteria. PTA over 0.5/1/2/4 kHz of ≥70 dB HL and ≤100 dB HL and sentence recognition test scores ≤60% at screening
  7. Patients with intact vestibular function in at least one ear (non-study ear) as measured by rotary chair vestibulo-ocular reflex or caloric nystagmography, or vestibular evoked myogenic potential (VEMP)
  8. MRI scan within 3 months or at screening to confirm suitability for inner ear surgery
  9. Ability to understand the study and provide informed consent; willingness to comply with the protocol

Exclusion Criteria:

  1. Patients with hearing loss caused by genetic/developmental disorders, e.g., cochlea aplasia or autoimmune ear disease
  2. Patients with existing conductive hearing loss or mixed hearing loss
  3. Patients with cochlear implants or past cochlear implant in the candidate 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, such as oncology patients on platinum-based chemotherapy
  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
Open or close this module Contacts/Locations
Central Contact Person: Novartis Pharmaceuticals
Telephone: 1-888-669-6682
Central Contact Backup: Novartis Pharmaceuticals
Study Officials: Novartis Pharmaceuticals
Study Director
Novartis Pharmaceuticals
Locations: United States, California
Novartis Investigative Site
San Francisco, California, United States, 94115
United States, Kansas
Novartis Investigative Site
Kansas City, Kansas, United States
United States, Maryland
Novartis Investigative Site
Baltimore, Maryland, United States, 21287
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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