First in Human Safety Study of FX-322 in Adults Undergoing Cochlear Implantation

This is a phase 1 safety study performed in male or female adult participants with an established diagnosis of severe to profound sensorineural hearing loss that meets the criteria for cochlear implantation and the participant has already chosen to undergo cochlear implant surgery.

Study Overview

• Status: Completed
• Conditions: Hearing Loss
• Intervention / Treatment: Drug: FX-322; Drug: Placebo

Detailed Description

Approximately 12 participants will be enrolled in the study. Assessed will be safety and tolerability of FX-322 administered by intratympanic injection. Also assessed will be the FX-322 concentration in cochlear fluid (perilymph), the pharmacokinetic (PK) profile of FX-322 to determine the systemic exposure to FX-322.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia
      • Royal Victoria Eye and Ear Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male or female adult subjects with an established diagnosis of severe to profound sensorineural hearing loss audiometric threshold of 80 dB HL or poorer at 500 Hz that meets the criteria for cochlear implantation and the subject has already chosen to undergo cochlear implant surgery.
2. Willingness and ability to comply with scheduled visits, ear examination, drug administration plan, auditory and laboratory tests, study restrictions, and all study procedures.

Other protocol-defined inclusion criteria may apply

Exclusion Criteria:

1. Persistent perforation of tympanic membrane or other tympanic membrane disorder that would interfere with the delivery and safety assessment of an intra -tympanic medication or reasonably be suspected to affect tympanic membrane healing after injection.
2. Any conductive component defined as air-bone gaps >10 dB at two or more frequencies.

Other protocol-defined exclusion criteria may apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Active; Subjects will receive FX-322 as an intratympanic injection	Drug: FX-322; intratympanic injection
Placebo Comparator: Placebo; Subjects will receive Placebo as an intratympanic injection	Drug: Placebo; intratympanic injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events (Safety and Tolerability) of FX-322.	To assess the incidence of adverse safety events (vertigo, tinnitus, perforation) including severe adverse events.	Treatment-emergent adverse events will be assessed over a several hour to two week period

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma Pharmacokinetics	Drug exposure in the systemic circulation by evaluating Cmax and AUC Drug exposure evaluated by area under the plasma concentration versus time curve (AUC)	Systemic exposure will be evaluated over a 72 hour period
Plasma Pharmacokinetics	Peak Plasma Concentration (Cmax)	Within a 24 hour period after injection
Cochlear Perilymph Pharmacokinetics	single time point measurement in each patient of drug in cochlear perilymph	within a 24 hour period after injection

Collaborators and Investigators

• Sponsor: Frequency Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2017
• Primary Completion (Actual): December 12, 2017
• Study Completion (Actual): January 18, 2018

Study Registration Dates

• First Submitted: September 15, 2017
• First Submitted That Met QC Criteria: September 28, 2017
• First Posted (Actual): October 3, 2017

Study Record Updates

• Last Update Posted (Actual): April 12, 2018
• Last Update Submitted That Met QC Criteria: April 11, 2018
• Last Verified: April 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Otorhinolaryngologic Diseases; Ear Diseases; Sensation Disorders; Hearing Disorders; Hearing Loss
• Other Study ID Numbers: FX-322-103

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No