A Clinical Trial of Anakinra for Steroid-Resistant Autoimmune Inner Ear Disease

This study has been completed.
National Institute on Deafness and Other Communication Disorders (NIDCD)
Information provided by (Responsible Party):
Andrea Vambutas, North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:
First received: December 28, 2010
Last updated: March 23, 2015
Last verified: March 2015

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.

Condition Intervention Phase
Sensorineural Hearing Loss
Autoimmune Inner Ear Disease
Drug: Anakinra
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/II Open-label Study of the Effects of Anakinra in Corticosteroid-resistant Subjects With Autoimmune Inner Ear Disease

Resource links provided by NLM:

Further study details as provided by North Shore Long Island Jewish Health System:

Primary 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 ] [ Designated as safety issue: Yes ]
    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.

Secondary Outcome Measures:
  • To Assess the Safety and Tolerability of a Three Month (84 Day) Course of Anakinra in Corticosteroid Resistant Patients With Autoimmune Inner Ear Disease. [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]

Enrollment: 13
Study Start Date: June 2011
Study Completion Date: September 2014
Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single Arm-Open Label
Single Arm-Open Label use of Anakinra
Drug: Anakinra
100mg of anakinra administered by a subcutaneous injection for 84 consecutive days.
Other Name: Kineret

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.


Ages Eligible for Study:   13 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01267994

United States, New York
North Shore-LIJ Hearing and Speech Center
New Hyde Park, New York, United States, 11040
Sponsors and Collaborators
Andrea Vambutas
National Institute on Deafness and Other Communication Disorders (NIDCD)
Principal Investigator: Andrea Vambutas, MD North Shore-LIJ Health System
  More Information

Additional Information:
Responsible Party: Andrea Vambutas, Professor of Otolaryngology & Molecular Medicine, North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier: NCT01267994     History of Changes
Other Study ID Numbers: 1R21DC011827-01, R33DC011827
Study First Received: December 28, 2010
Results First Received: September 16, 2014
Last Updated: March 23, 2015
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by North Shore Long Island Jewish Health System:
Autoimmune Inner Ear Disease
Immune Mediated Hearing Loss
Sudden Sensorineural Hearing Loss
Meniere's Disease

Additional relevant MeSH terms:
Ear Diseases
Hearing Loss
Hearing Loss, Sensorineural
Labyrinth Diseases
Hearing Disorders
Nervous System Diseases
Neurologic Manifestations
Otorhinolaryngologic Diseases
Sensation Disorders
Signs and Symptoms

ClinicalTrials.gov processed this record on October 13, 2015