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

NCT01267994 | Completed Phase 1 Results DMC

• 2 other identifiers: 1R21DC011827-01R33DC011827
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (1)

• To Assess the Potential Efficacy of Anakinra in Improving Hearing Thresholds in Corticosteroid-resistant Patients With Autoimmune Inner Ear Disease

  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.

  180 days

Secondary Outcomes (1)

• Number of Serious Adverse Events Reported

  84 days

Study Arms (1)

Single Arm-Open Label

EXPERIMENTAL

Single Arm-Open Label use of Anakinra

Drug: Anakinra

Interventions

Anakinra DRUG

100mg of anakinra administered by a subcutaneous injection for 84 consecutive days.

Also known as: Kineret

Single Arm-Open Label

Eligibility Criteria

• Age: 13 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Andrea Vambutas: lead
• National Institute on Deafness and Other Communication Disorders (NIDCD): collaborator

Study Sites (1)

North Shore-LIJ Hearing and Speech Center

New Hyde Park, New York, 11040, United States

Location

Related Publications (1)

• 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.

  PMID: 25133431 RESULT

Related Links

• Clinical trials at the Feinstein Institute for Medical Research

MeSH Terms

Conditions

Hearing Loss, Sensorineural; Hearing Loss, Sudden; Meniere Disease

Interventions

Interleukin 1 Receptor Antagonist Protein

Condition Hierarchy (Ancestors)

Hearing Loss; Hearing Disorders; Ear Diseases; Otorhinolaryngologic Diseases; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Endolymphatic Hydrops; Labyrinth Diseases

Intervention Hierarchy (Ancestors)

Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Andrea Vambutas, MD, FACS
• Organization: North Shore-LIJ Health System

vambutas@nshs.edu

718-470-7748

Study Officials

• Andrea Vambutas, MD

  Northwell Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Otolaryngology & Molecular Medicine

Study Record Dates

• First Submitted: December 28, 2010
• First Posted: December 29, 2010
• Study Start: June 1, 2011
• Primary Completion: September 1, 2014
• Study Completion: September 1, 2014
• Last Updated: January 10, 2018
• Results First Posted: April 6, 2015

Record last verified: 2017-12

Locations

US (1)