Autoimmune Inner Ear DiseasePhase 2NCT03587701
Effects of Anakinra in Subjects With Corticosteroid-resistant/Intolerant Meniere's Disease and Autoimmune Inner Ear Disease
A Phase 2 study testing Anakinra 100Mg/0.67Ml Inj Syringe (Period 1) and Anakinra 100Mg/0.67Ml Inj Syringe (Period 2) in people with Autoimmune Inner Ear Disease and Meniere's Disease, run by Northwell Health at 1 site in United States.
Trial summary
A Phase II randomized, placebo controlled study design of anakinra (Kineret) in corticosteroid-resistant or intolerant Meniere's disease (CR-MD)and corticosteroid-resistant or intolerant autoimmune inner ear disease (CR-AIED) patients. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Autoimmune Inner Ear Disease, Meniere's Disease
- What is being tested?
- Anakinra 100Mg/0.67Ml Inj Syringe (Period 1) and Anakinra 100Mg/0.67Ml Inj Syringe (Period 2)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 57 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Jan 2019to 31 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- meet diagnostic criteria for Autoimmune Inner Ear Disease (AIED) with active deterioration in one ear as defined below
- SNHL of greater than 30dB at one or more frequencies in the active ear. The contralateral ear may have any hearing threshold that is either stable or worsening, but may not exhibit improvement in response to corticosteroids even if the more active ear declines. Active deterioration is defined as a 15dB decline at one frequency (excluding 250 or 8kHz as a sole indicator) on their audiogram, or 10dB at 2+ frequencies developing in > or equal 3 days but < or equal 90 days, or if the hearing loss evolved in less than 3 days, the patient displayed features suggestive of an autoimmune disorder
- Capable of understanding and giving informed consent
- Have 2 sequential audiograms following the decline in hearing >or equal 28 days apart with no demonstrable improvement in hearing following corticosteroid therapy (<or equal 5dB of improvement at any individual frequency (250, 500, 1000, 2000 and 4000Hz) and <12% improvement in WRS)
- Patients must have been treated with oral or IT prednisone with no demonstrable improvement or attempted to be treated and deemed intolerant. Steroid intolerance is defined as unable or unwilling to take corticosteroids because of a prior untoward effect or a current medical condition that would negatively be impacted by steroid use
- Must have completed steroid therapy, including any intratympanic steroid therapy
Show all 10Show fewer
- Meniere's disease defined according to the AAO criteria, with the additional requirement of corticosteroid resistance AND no new diuretics may be instituted or dose increased from 28 days prior to trial enrollment
- Patients must be fluent in English as all word recognition scores are based on testing in English
- Patients may have other autoimmune disease(s) Females of childbearing potential, must be practicing a method of birth control considered effective and medically acceptable by the investigator for a minimum of 2 months prior to the study and at least 2 months after last administration of study drug
- Males similarly must be practicing a method of birth control considered effective and medically acceptable by the investigator for a minimum of 2 months prior to the study and at least 2 months after last administration of study drug
Who cannot join
- Evidence of retrocochlear pathology (vestibular schwannoma) or inner ear malformation (Mondini Malformation or Enlarged Vestibular Aqueduct) based on imaging
- Prior treatment with gentamicin for Meniere's Disease
- Most recent decline in hearing occurring >90 days prior to trial enrollment
- Positive test for Muckle-Wells mutation
- Concurrently receiving methotrexate, TNF-antagonist therapy or any other immunosuppressive therapy
- Steroid-dependent hearing loss
Show all 21Show fewer
- Any immunodeficiency syndrome
- Active or chronic infections
- Currently receiving, or having received treatment for a malignancy in the past 3 years
- Chronic renal insufficiency (a creatinine clearance of <49mL/min) or chronic renal failure
- Neutropenia prior to treatment with anakinra
- Receipt of live vaccine <3 months prior to enrollment
- Previous treatment with an IL-1 antagonist for any clinical indication
- First-degree relative with a diagnosis of a CAPS disease
- History of active narcotic abuts, including prescription narcotics
- Pregnant or lactating females
- Non-English speaking patients
- Seropositive for Hepatitis B, C, HIV or tuberculosis on screening, with the exception of presence of antibodies to Hepatitis B in subjects reporting prior vaccination, and presence of positive skin testing for TB in subjects who received BCG in the past
- History of suicidal ideation, significant depression that resulted in hospitalization, or answers "yes" during C-SSR screening
- Prior participation in any type of clinical trial using a medication that inhibits interleukin-1 activity
- Known hypersensitivity to E.coli derived products
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Northwell Health, Hearing& Speech Center
New Hyde Park, New York, 11042
Andrea Vambutas, MD, 718-470-7550, avambuta@northwell.edu
Ginny Mullooly, RN, 718-470-7974, gmullool@northwell.edu
About this study
Official title: A Phase II Placebo Controlled Study of the Effects of Anakinra (Kineret) in Subjects With Corticosteroid-Resistant/Intolerant Meniere's Disease (CR-MD) and Corticosteroid-Resistant/Intolerant Autoimmune Inner Ear Disease Disease (CR-AIED)
A Phase II randomized, placebo controlled study design of anakinra (Kineret) in corticosteroid-resistant or intolerant Meniere's disease (CR-MD)and corticosteroid-resistant or intolerant autoimmune inner ear disease (CR-AIED) patients. Patients will be randomized by a 2:1 allocation to anakinra or placebo for 42 continuous days. After day 42, a second placebo-controlled period will begin for an additional 42 days. This will be followed by a 264 day observation period, during which, hearing declines may be re-treated with anakinra after 30 days following the initial drug.
Read the detailed description
This study will be evaluating whether or not anakinra (kineret) is superior to placebo for improving sustained hearing thresholds in patients with CR or intolerant MD and CR or intolerant AIED. Final enrollment is contingent on an acute decline in hearing in the active ear and 2 sequential audiograms greater than or equal to 28 days apart that shows a stable or declining hearing threshold and word recognition score not improved with corticosteroid therapy. Any patient that attempted to be treated with steroid therapy and was deemed intolerant will also be considered for the trial. Anakinra (kineret) 100mg/0.67ml prefilled syringes or placebo injection will be randomized at 2:1 allocation and will be self-administered by the patient daily for 42 days. After day 42, a second placebo-controlled period will begin for an additional 42 days (all patients will receive anakinra for either 42 or 84 days depending on the randomization assignment). A 264 day observation period will begin after day 84, during which the option to retreat with anakinra will be offered to those patients that responded to the therapy in the first 84 day period.
- Conditions
- Autoimmune Inner Ear Disease, Meniere's Disease
- Being tested
- Anakinra 100Mg/0.67Ml Inj Syringe (Period 1), Placebo injection (Period 1), Anakinra 100Mg/0.67Ml Inj Syringe (Period 2), Placebo injection (Period 2)
- Medical subject headings
- Meniere Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anakinra 100Mg/0.67Ml Inj Syringe (Period 1)Drug
pre-filled syringes of anakinra 100mg/0.67mg will be provided and will be self-administered by patients daily during first 42 days of treatment (Period 1)
Placebo injection (Period 1)Drug
pre-filled matching placebo injections will be provided and will be self-administered by patients daily during first 42 days of treatment (Period 1)
Anakinra 100Mg/0.67Ml Inj Syringe (Period 2)Drug
pre-filled syringes of anakinra 100mg/0.67mg will be provided and will be self-administered by patients daily during next 42 days of treatment (Period 2)
Placebo injection (Period 2)Drug
pre-filled matching placebo injections will be provided and will be self-administered by patients daily during next 42 days of treatment (Period 2)
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | This group will be randomized to receive intervention of 42 consecutive days of anakinra (100mg/0.67ml) in period 1 followed by an additional 42 consecutive days of anakinra (100mg/0.67ml) in period 2 Receives: Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 1); Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 2) |
| Group B | Experimental | This group will be randomized to receive intervention of 42 consecutive days of anakinra (100 mg/0.67ml) in period 1 followed by 42 consecutive days of placebo in period 2 Receives: Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 1); Drug: Placebo injection (Period 2) |
| Group C | Experimental | This group will be randomized to receive intervention of placebo for 42 consecutive days in period 1 followed by 42 consecutive days of anakinra (100mg/0.67ml) in period 2 Receives: Drug: Placebo injection (Period 1); Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 2) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sustained improvement in hearing thresholds
Determine whether those treated with anakinra demonstrate a sustained improvement in hearing thresholds compared with their pre-anakinra treatment threshold. Improvement will be defined by an improvement of 15dB at 1 frequency or 10dB at 2 contiguous frequencies with no greater than a 5dB worsening all remaining frequencies
Measured: Hearing improvement from pre-treatment thresholds at day 28
Sustained improvement in hearing thresholds
threshold. Improvement will be defined by an improvement of 15dB at 1 frequency or 10dB at 2 contiguous frequencies with no greater than a 5dB worsening all remaining frequencies
Measured: Hearing improvement from pre-treatment thresholds at day 42
Secondary outcomes (2)
Improved Word Recognition
Determine whether those treated with anakinra demonstrate a sustained improvement in word recognition score compared with their pre-anakinra treatment threshold. Improvement will be defined by an improvement of greater than 12% change in value
Measured: at day 28
Improved Word Recognition
Determine whether those treated with anakinra demonstrate a sustained improvement in word recognition score compared with their pre-anakinra treatment threshold. Improvement will be defined by an improvement of greater than 12% change in value
Measured: at day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2018
- Study start3 Jan 2019
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autoimmune Inner Ear DiseaseStudies of the same condition, open and under way.
- More trials run by Northwell HealthThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study is a randomized, placebo-controlled design with a 2:1 allocation of anakinra or placebo for the first 42 continuous days. After day 42, a second placebo-controlled period will begin for an additional 42 days.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 57 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Northwell Health
- Sponsor type
- University, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Responsible party
- Sponsor
- Investigators
- Andrea Vambutas, MD, Principal investigator, Northwell Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03587701
- Sponsor's protocol ID
- 18-0195
- Status last verified
- Apr 2026
- First submitted
- 12 Jun 2018
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
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 25133431Pathak S, Goldofsky E, Vivas EX, Bonagura VR, Vambutas A. IL-1beta is overexpressed and aberrantly regulated in corticosteroid nonresponders with autoimmune inner ear disease. J Immunol. 2011 Feb 1;186(3):1870-9. doi: 10.4049/jimmunol.1002275. Epub 2011 Jan 3.
PubMed 21199898
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Placebo Controlled Study of the Effects of Anakinra (Kineret) in Subjects With Corticosteroid-Resistant/Intolerant Meniere's Disease (CR-MD) and Corticosteroid-Resistant/Intolerant Autoimmune Inner Ear Disease Disease (CR-AIED)
- ClinicalTrials.gov ID
- NCT03587701
- Acronym / sponsor ID
- 18-0195
- Conditions
- Autoimmune Inner Ear Disease, Meniere's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 57 participants (estimated)
- Lead sponsor
- Northwell HealthUniversity, hospital or other
- Collaborators
- Swedish Orphan Biovitrum
- Study start
- 3 Jan 2019 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 16 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase II randomized, placebo controlled study design of anakinra (Kineret) in corticosteroid-resistant or intolerant Meniere's disease (CR-MD)and corticosteroid-resistant or intolerant autoimmune inner ear disease (CR-AIED) patients. Patients will be randomized by a 2:1 allocation to anakinra or placebo for 42 continuous days. After day 42, a second placebo-controlled period will begin for an additional 42 days. This will be followed by a 264 day observation period, during which, hearing declines may be re-treated with anakinra after 30 days following the initial drug.
Detailed description
This study will be evaluating whether or not anakinra (kineret) is superior to placebo for improving sustained hearing thresholds in patients with CR or intolerant MD and CR or intolerant AIED. Final enrollment is contingent on an acute decline in hearing in the active ear and 2 sequential audiograms greater than or equal to 28 days apart that shows a stable or declining hearing threshold and word recognition score not improved with corticosteroid therapy. Any patient that attempted to be treated with steroid therapy and was deemed intolerant will also be considered for the trial. Anakinra (kineret) 100mg/0.67ml prefilled syringes or placebo injection will be randomized at 2:1 allocation and will be self-administered by the patient daily for 42 days. After day 42, a second placebo-controlled period will begin for an additional 42 days (all patients will receive anakinra for either 42 or 84 days depending on the randomization assignment). A 264 day observation period will begin after day 84, during which the option to retreat with anakinra will be offered to those patients that responded to the therapy in the first 84 day period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
- Model description
- This study is a randomized, placebo-controlled design with a 2:1 allocation of anakinra or placebo for the first 42 continuous days. After day 42, a second placebo-controlled period will begin for an additional 42 days.
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 1), Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 2)This group will be randomized to receive intervention of 42 consecutive days of anakinra (100mg/0.67ml) in period 1 followed by an additional 42 consecutive days of anakinra (100mg/0.67ml) in period 2 |
| Group B | Experimental | Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 1), Drug: Placebo injection (Period 2)This group will be randomized to receive intervention of 42 consecutive days of anakinra (100 mg/0.67ml) in period 1 followed by 42 consecutive days of placebo in period 2 |
| Group C | Experimental | Drug: Placebo injection (Period 1), Drug: Anakinra 100Mg/0.67Ml Inj Syringe (Period 2)This group will be randomized to receive intervention of placebo for 42 consecutive days in period 1 followed by 42 consecutive days of anakinra (100mg/0.67ml) in period 2 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- meet diagnostic criteria for Autoimmune Inner Ear Disease (AIED) with active deterioration in one ear as defined below
- SNHL of greater than 30dB at one or more frequencies in the active ear. The contralateral ear may have any hearing threshold that is either stable or worsening, but may not exhibit improvement in response to corticosteroids even if the more active ear declines. Active deterioration is defined as a 15dB decline at one frequency (excluding 250 or 8kHz as a sole indicator) on their audiogram, or 10dB at 2+ frequencies developing in > or equal 3 days but < or equal 90 days, or if the hearing loss evolved in less than 3 days, the patient displayed features suggestive of an autoimmune disorder
- Capable of understanding and giving informed consent
- Have 2 sequential audiograms following the decline in hearing >or equal 28 days apart with no demonstrable improvement in hearing following corticosteroid therapy (<or equal 5dB of improvement at any individual frequency (250, 500, 1000, 2000 and 4000Hz) and <12% improvement in WRS)
- Patients must have been treated with oral or IT prednisone with no demonstrable improvement or attempted to be treated and deemed intolerant. Steroid intolerance is defined as unable or unwilling to take corticosteroids because of a prior untoward effect or a current medical condition that would negatively be impacted by steroid use
Key exclusion criteria (21 listed)
- Evidence of retrocochlear pathology (vestibular schwannoma) or inner ear malformation (Mondini Malformation or Enlarged Vestibular Aqueduct) based on imaging
- Prior treatment with gentamicin for Meniere's Disease
- Most recent decline in hearing occurring >90 days prior to trial enrollment
- Positive test for Muckle-Wells mutation
- Concurrently receiving methotrexate, TNF-antagonist therapy or any other immunosuppressive therapy
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 |
|---|---|---|
| Anakinra 100Mg/0.67Ml Inj Syringe (Period 1)pre-filled syringes of anakinra 100mg/0.67mg will be provided and will be self-administered by patients daily during first 42 days of treatment (Period 1) | Drug | Group A, Group B |
| Placebo injection (Period 1)pre-filled matching placebo injections will be provided and will be self-administered by patients daily during first 42 days of treatment (Period 1) | Drug | Group C |
| Anakinra 100Mg/0.67Ml Inj Syringe (Period 2)pre-filled syringes of anakinra 100mg/0.67mg will be provided and will be self-administered by patients daily during next 42 days of treatment (Period 2) | Drug | Group A, Group C |
| Placebo injection (Period 2)pre-filled matching placebo injections will be provided and will be self-administered by patients daily during next 42 days of treatment (Period 2) | Drug | Group B |
Primary outcome
Sustained improvement in hearing thresholds
Time frame: Hearing improvement from pre-treatment thresholds at day 28
Determine whether those treated with anakinra demonstrate a sustained improvement in hearing thresholds compared with their pre-anakinra treatment threshold. Improvement will be defined by an improvement of 15dB at 1 frequency or 10dB at 2 contiguous frequencies with no greater than a 5dB worsening all remaining frequencies
Sustained improvement in hearing thresholds
Time frame: Hearing improvement from pre-treatment thresholds at day 42
threshold. Improvement will be defined by an improvement of 15dB at 1 frequency or 10dB at 2 contiguous frequencies with no greater than a 5dB worsening all remaining frequencies
2 secondary outcomes
Improved Word Recognition
Time frame: at day 28
Improved Word Recognition
Time frame: at day 42
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- 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.
- Results publication: PubMed 21199898Pathak S, Goldofsky E, Vivas EX, Bonagura VR, Vambutas A. IL-1beta is overexpressed and aberrantly regulated in corticosteroid nonresponders with autoimmune inner ear disease. J Immunol. 2011 Feb 1;186(3):1870-9. doi: 10.4049/jimmunol.1002275. Epub 2011 Jan 3.
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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.
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 2 study testing Anakinra 100Mg/0.67Ml Inj Syringe (Period 1) and Anakinra 100Mg/0.67Ml Inj Syringe (Period 2) in people with Autoimmune Inner Ear Disease and Meniere's Disease, run by Northwell Health 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 10 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 15 Apr 2026. 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ginny Mullooly, RN (718-470-7974, gmullool@northwell.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sustained improvement in hearing thresholds (time frame: Hearing improvement from pre-treatment thresholds at day 28). The study lists 2 primary outcomes in all. 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 NCT03587701, which also lists 2 linked publications.
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 15 Apr 2026.