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.
Full Title of Study: “A Phase I/II Open-label Study of the Effects of Anakinra in Corticosteroid-resistant Subjects With Autoimmune Inner Ear Disease”
- Study Type: Interventional
- Study Design
- Allocation: N/A
- Intervention Model: Single Group Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: September 2014
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.
- Drug: Anakinra
- 100mg of anakinra administered by a subcutaneous injection for 84 consecutive days.
Arms, Groups and Cohorts
- Experimental: Single Arm-Open Label
- Single Arm-Open Label use of Anakinra
Clinical Trial Outcome Measures
- To Assess the Potential Efficacy of Anakinra in Improving Hearing Thresholds in Corticosteroid-resistant Patients With Autoimmune Inner Ear Disease
- Time Frame: 180 days
- The primary endpoint is to determine whether those treated with anakinra for 84 days demonstrate an improved hearing threshold compared with their pre-anakinra-treatment threshold. Audiometric thresholds will be compared to those treated with a prolonged corticosteroid taper and those that elect for no further treatment. The durability of the response will be measured over a total of 180 days.
- Number of Serious Adverse Events Reported
- Time Frame: 84 days
- To assess the number of Serious Adverse Events reported in any subject that received at least one injection dose of anakinra
Participating in This Clinical Trial
- 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
Gender Eligibility: All
Minimum Age: 13 Years
Maximum Age: 75 Years
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- Andrea Vambutas
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Provider of Information About this Clinical Study
- Sponsor-Investigator: Andrea Vambutas, Professor of Otolaryngology & Molecular Medicine – Northwell Health
- Overall Official(s)
- Andrea Vambutas, MD, Principal Investigator, Northwell Health
Citations Reporting on Results
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.
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