Sensorineural Hearing LossPhase 1/2NCT01267994
A Clinical Trial of Anakinra for Steroid-Resistant Autoimmune Inner Ear Disease
A Phase 1/2 study testing Anakinra in people with Sensorineural Hearing Loss and Autoimmune Inner Ear Disease, run by Andrea Vambutas at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 13 to 75 years
- Conditions the study is for
- Sensorineural Hearing Loss, Autoimmune Inner Ear Disease
- What is being tested?
- Anakinra
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2011to Sep 2014
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Age over 75, or less than 13
- Neutropenia
- Renal insufficiency
- Pregnant females
- Unilateral hearing loss
- Patients with any immunodeficiency syndrome
Show all 12Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
North Shore-LIJ Hearing and Speech Center
New Hyde Park, New York, 11040
About this study
Official title: A Phase I/II Open-label Study of the Effects of Anakinra in Corticosteroid-resistant Subjects With Autoimmune Inner Ear Disease
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.
Read the 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.
- Being tested
- Anakinra
- Keywords
- Steroid, Steroid-Resistant, Autoimmune Inner Ear Disease, Immune Mediated Hearing Loss, Sudden Sensorineural Hearing Loss, Meniere's Disease, Autoimmune, Hearing
- Medical subject headings
- Hearing Loss, Sensorineural, Hearing Loss, Sudden, Meniere Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AnakinraDrug
Also called Kineret
100mg of anakinra administered by a subcutaneous injection for 84 consecutive days.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm-Open Label | Experimental | Single Arm-Open Label use of Anakinra Receives: Drug: Anakinra |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 180 days
Secondary outcomes (1)
Number of Serious Adverse Events Reported
To assess the number of Serious Adverse Events reported in any subject that received at least one injection dose of anakinra
Measured: 84 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2010 (estimated)
- Study startJun 2011
- Primary completionSep 2014
- Study completionSep 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sensorineural Hearing LossStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Andrea Vambutas
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Responsible party
- Sponsor-investigator, Andrea Vambutas, Northwell Health
- Investigators
- Andrea Vambutas, MD, Principal investigator, Northwell Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01267994
- Sponsor's protocol ID
- 1R21DC011827-01
- Other IDs
- R33DC011827
- Status last verified
- Dec 2017
- First submitted
- 28 Dec 2010
- Last updated
- 10 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 25133431
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Open-label Study of the Effects of Anakinra in Corticosteroid-resistant Subjects With Autoimmune Inner Ear Disease
- ClinicalTrials.gov ID
- NCT01267994
- Acronym / sponsor ID
- 1R21DC011827-01
- Keywords
- Steroid, Steroid-Resistant, Autoimmune Inner Ear Disease, Immune Mediated Hearing Loss, Sudden Sensorineural Hearing Loss, Meniere's Disease, Autoimmune, Hearing
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Andrea VambutasUniversity, hospital or other
- Collaborators
- National Institute on Deafness and Other Communication Disorders (NIDCD)
- Study start
- Jun 2011
- Primary completion
- Sep 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 29 Dec 2010 (estimated)
- Results
- Posted 6 Apr 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm-Open Label | Experimental | Drug: AnakinraSingle Arm-Open Label use of Anakinra |
Who may qualify
- Ages
- 13 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (12 listed)
- Age over 75, or less than 13
- Neutropenia
- Renal insufficiency
- Pregnant females
- Unilateral hearing loss
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AnakinraAlso: Kineret100mg of anakinra administered by a subcutaneous injection for 84 consecutive days. | Drug | Single Arm-Open Label |
Primary outcome
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.
1 secondary outcome
Number of Serious Adverse Events Reported
Time frame: 84 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 25133431Vambutas 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.
- Clinical trials at the Feinstein Institute for Medical ResearchLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrea Vambutas, MD principal investigator
Northwell Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Anakinra in people with Sensorineural Hearing Loss and Autoimmune Inner Ear Disease, run by Andrea Vambutas at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Apr 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To Assess the Potential Efficacy of Anakinra in Improving Hearing Thresholds in Corticosteroid-resistant Patients With Autoimmune Inner Ear Disease (time frame: 180 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01267994, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2018.