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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01267994
Recruitment Status : Completed
First Posted : December 29, 2010
Results First Posted : April 6, 2015
Last Update Posted : January 10, 2018
National Institute on Deafness and Other Communication Disorders (NIDCD)
Information provided by (Responsible Party):
Andrea Vambutas, Northwell Health

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.

Condition or disease Intervention/treatment Phase
Sensorineural Hearing Loss Autoimmune Inner Ear Disease Drug: Anakinra Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : June 2011
Actual Primary Completion Date : September 2014
Actual Study Completion Date : September 2014

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   13 Years to 75 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01267994

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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)
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Principal Investigator: Andrea Vambutas, MD Northwell Health
Additional Information:
Publications of Results:
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Responsible Party: Andrea Vambutas, Professor of Otolaryngology & Molecular Medicine, Northwell Health Identifier: NCT01267994    
Other Study ID Numbers: 1R21DC011827-01 ( U.S. NIH Grant/Contract )
R33DC011827 ( U.S. NIH Grant/Contract )
First Posted: December 29, 2010    Key Record Dates
Results First Posted: April 6, 2015
Last Update Posted: January 10, 2018
Last Verified: December 2017
Keywords provided by Andrea Vambutas, Northwell Health:
Autoimmune Inner Ear Disease
Immune Mediated Hearing Loss
Sudden Sensorineural Hearing Loss
Meniere's Disease
Additional relevant MeSH terms:
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Hearing Loss
Hearing Loss, Sensorineural
Ear Diseases
Labyrinth Diseases
Hearing Disorders
Otorhinolaryngologic Diseases
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Interleukin 1 Receptor Antagonist Protein
Antirheumatic Agents