Clinical trial NCT03616223: FX-322 in Sensorineural Hearing Loss
Official title: A Phase 1/2 Randomized, Double-blind, Placebo-controlled Single Dose Study at Two Dose Levels of FX-322 Administered by Intratympanic Injection in Adults With Stable Sensorineural Hearing Loss
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Sensorineural Hearing Loss
Keywords: Intratympanic Administration
Hearing Restoration
Lead sponsor: Frequency Therapeutics
Sponsor class: INDUSTRY
First posted: 2018-08-06
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a phase 1/2 study of FX-322 at two dose levels compared to placebo in male and female adults otherwise healthy with stable sensorineural hearing loss.

Detailed description: Sensorineural hearing loss (SNHL) accounts for about 90% of all cases of hearing loss. The study will assess the safety of FX-322 (laduviglusib and sodium valproate) given as a single intratympanic injection in subjects with a medical history of sensorineural hearing loss that is associated with noise exposure or sudden hearing loss. Safety will be evaluated both systemically (lab and clinical monitoring) and locally (otoscopy and audiometry) in 24 subjects, and a blood PK profile of FX-322 will also be determined.

Eligibility criteria: Inclusion Criteria:

1. Adult aged 18-65 years.
2. Established diagnosis of stable sensorineural hearing loss (no changes of 10 dB or more at any frequency) by standard audiometric measures for \>6 months.
3. Documented medical history consistent with hearing loss being caused by noise exposure or sudden sensorineural hearing loss (documented audiogram at least 6 months prior to screening required).
4. Female subjects must be of non-childbearing potential or will need to utilize two methods of highly effective contraception during the study participation (e.g. hormonal contraception or an intrauterine device and condoms) or remain abstinent. Male subjects should use condoms with spermicide during the course of the study or remain abstinent. Subjects should not donate sperm or ova during the study period.

Exclusion Criteria:

1. Perforation of tympanic membrane or other tympanic membrane disorders that would interfere with the delivery and safety assessment of an intratympanic medication or reasonably be suspected to affect tympanic membrane healing after injection in either ear. This includes a current tympanostomy tubes.
2. Any conductive hearing loss of 10 dB or more at two or more frequencies in either ear.
3. A pure tone average of 70 dB or greater at 500Hz, 1000Hz, 2000Hz, and 4000Hz in the ear to be injected.
4. Active chronic middle ear disease or a history of major middle ear surgery, as an adult, in the ear to be injected.
5. Subject has had an intratympanic injection in either ear within 6 months of the screening visit.
6. History of clinically significant vestibular symptoms at the discretion of the investigator.
7. History of clinically significant systemic autoimmune disease (e.g. rheumatoid arthritis, Sjogren's syndrome, multiple sclerosis, psoriasis).
8. History of head or neck radiation treatment or exposure.
9. History of substance abuse within 2 years of the Screening Visit.
10. Positive urine pregnancy test or breast-feeding.
11. Any known factor, condition or disease that, in the view of the investigator, might interfere with treatment compliance, study conduct or interpretation of the results such as psychiatric disease or suicidal tendencies.

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Event(s) (TEAEs)
timeFrame: Baseline through Day 90
Secondary outcomes:
measure: Cmax
timeFrame: Data points taken pre-dose and 0.5, 1, 2, 4, 8, 24 hours post-dose
measure: Tmax
timeFrame: Data points taken pre-dose and 0.5, 1, 2, 4, 8, 24 hours post-dose
measure: AUClast
timeFrame: Data points taken pre-dose and 0.5, 1, 2, 4, 8, 24 hours post-dose
measure: t1/2
timeFrame: Data points taken pre-dose and 0.5, 1, 2, 4, 8, 24 hours post-dose
measure: CL/F
timeFrame: Data points taken pre-dose and 0.5, 1, 2, 4, 8, 24 hours post-dose
Other outcomes:
Locations:
city: San Antonio
country: United States
facility: Worldwide Clinical Trials
state: Texas
Study officials:
affiliation: Worldwide Clinical Trials
name: George Atiee, MD
role: PRINCIPAL_INVESTIGATOR
References: