Clinical trial NCT01267994: A Clinical Trial of Anakinra for Steroid-Resistant Autoimmune Inner Ear Disease
Official title: A Phase I/II Open-label Study of the Effects of Anakinra in Corticosteroid-resistant Subjects With Autoimmune Inner Ear Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Sensorineural Hearing Loss
Autoimmune Inner Ear Disease
Keywords: Steroid
Steroid-Resistant
Autoimmune Inner Ear Disease
Immune Mediated Hearing Loss
Sudden Sensorineural Hearing Loss
Meniere's Disease
Autoimmune
Hearing
Lead sponsor: Northwell Health
Sponsor class: OTHER
Collaborators: name: National Institute on Deafness and Other Communication Disorders (NIDCD)
First posted: 2010-12-29
Enrollment: 13
Sex: ALL
Minimum age (years): 13
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine if Anakinra (an interleukin-1 receptor antagonist) can improve hearing thresholds in those patients with Autoimmune Inner Ear Disease (AIED) that did not respond to oral steroid therapy for a sudden decline in hearing. The patients to be enrolled will have recently completed a course of oral steroids and demonstrated no change in their audiometric thresholds following corticosteroid therapy.

Detailed description: Patients with immune mediated hearing loss (also known as autoimmune hearing loss) are typically treated with corticosteroids. Of those treated, approximately 60% respond, however, that response may be lost over time. Other therapies use to date have proven largely ineffectual in improving hearing. This study proposes a phase I open label clinical trial of Anakinra for corticosteroid-resistant patients to determine if this therapy is efficacious in hearing restoration.

Eligibility criteria: Inclusion Criteria:

* Bilateral sensorineural hearing loss with an active decline in hearing in one ear
* No audiometric improvement with 28-30 days of oral prednisone or other corticosteroid, including an initial dose of 60mg per day for 14 days
* Enrollment within 14 days of completion of corticosteroid therapy
* Age 13 years and older
* No evidence of neutropenia (low white blood cell count)
* No evidence of retrocochlear pathology (ie. acoustic neuroma/vestibular schwannoma)
* May have concurrent, systemic autoimmune disease

Exclusion Criteria:

* Age over 75, or less than 13
* Neutropenia
* Renal insufficiency
* Pregnant females
* Unilateral hearing loss
* Patients with any immunodeficiency syndrome
* Patients receiving methotrexate or any TNF (tumor necrosis factor) antagonist therapy
* Patients with chronic infections
* Patients treated for a malignancy within the past 3 years
* Patients with a latex allergy
* Patients with an inner ear anomaly
* Patients with retrocochlear pathology

Primary outcomes:
measure: To Assess the Potential Efficacy of Anakinra in Improving Hearing Thresholds in Corticosteroid-resistant Patients With Autoimmune Inner Ear Disease
timeFrame: 180 days
Secondary outcomes:
measure: Number of Serious Adverse Events Reported
timeFrame: 84 days
Other outcomes:
Locations:
city: New Hyde Park
country: United States
facility: North Shore-LIJ Hearing and Speech Center
state: New York
Study officials:
affiliation: Northwell Health
name: Andrea Vambutas, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Vambutas A, Lesser M, Mullooly V, Pathak S, Zahtz G, Rosen L, Goldofsky E. Early efficacy trial of anakinra in corticosteroid-resistant autoimmune inner ear disease. J Clin Invest. 2014 Sep;124(9):4115-22. doi: 10.1172/JCI76503. Epub 2014 Aug 18.