Rilonacept for Autoimmune Hearing Loss

Overseen By Cindy Cabrera

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Stanley Cohen

Must be taking: Prednisone

Must not be taking: Immunosuppressants, CYP3A4 inhibitors

Disqualifiers: Infections, Diabetes, Organ transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests rilonacept, a medication, to determine its effectiveness for autoimmune neurosensory hearing loss (ANSHL), where the immune system attacks the inner ear, causing hearing loss. Researchers aim to find out if rilonacept can improve hearing in individuals who have already shown some improvement with steroids like prednisone. Those who have experienced progressive hearing loss in both ears, confirmed by tests, and have improved after 30 days on prednisone may be suitable candidates. Participants will receive weekly injections for 24 weeks. As an Early Phase 1 trial, this research focuses on understanding how rilonacept works in people, offering participants a chance to be among the first to receive this treatment.

Do I need to stop my current medications for the trial?

The trial requires that you stop taking certain medications, including systemic immunosuppressants and specific drugs like strong CYP3A4 inhibitors and long-acting opiates, before starting the study. There are also restrictions on certain vaccines and steroid injections during the study.

Is there any evidence suggesting that rilonacept is likely to be safe for humans?

Research has shown that rilonacept has been tested in other conditions and is generally well-tolerated. In past studies, common side effects included upper respiratory issues like a stuffy nose and sore throat, as well as reactions at the injection site, such as pain or redness. Some patients also reported rashes or an upset stomach. Serious allergic reactions can occur, so signs like swelling or severe itching require immediate medical attention. The FDA has approved this treatment for other uses, indicating some established safety. However, since this trial is in its early stages, the researchers are still collecting complete safety information for autoimmune neurosensory hearing loss (ANSHL). ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for autoimmune neurosensory hearing loss involve steroids or other immunosuppressants that broadly dampen the immune response. But Rilonacept works differently, targeting a specific protein involved in inflammation called interleukin-1. This means it might reduce inflammation more precisely, potentially leading to fewer side effects compared to traditional broad-spectrum treatments. Researchers are excited because this targeted approach could offer a new way to manage the condition with potentially greater efficacy and improved safety.

What evidence suggests that rilonacept might be an effective treatment for ANSHL?

Research has shown that rilonacept, which participants in this trial will receive, can help reduce the recurrence of pericarditis, an inflammation of the heart's lining. In one study, only 7% of patients who took rilonacept experienced a recurrence of pericarditis, compared to a higher percentage before treatment. Rilonacept works by blocking proteins that cause inflammation. Although specific information about its use for autoimmune neurosensory hearing loss (ANSHL) is limited, its ability to reduce inflammation might also benefit this condition. Early results from similar inflammatory conditions suggest that rilonacept could be effective for ANSHL. ^{6 7 8 9 10}

Who Is on the Research Team?

Stanley B Cohen, MD

Principal Investigator

Metroplex Clinical Research Center

Are You a Good Fit for This Trial?

Inclusion Criteria

The presence of progressive sensironeural hearing loss greater than or equal to 30 dB in both ears at one or more frequencies (250, 500, 1000, 2000, 3000, 4000, 6000 or 8000 Hz) and idiopathic-based on clinical evaluation, blood tests, and radiographic imaging.

Documented improvement in hearing by audiogram after 30 days of treatment with high dose prednisone 40-60 mg/d. Improvement is defined by 10 dB improvement in pure tone average (500-3000 dB) or an improvement in word identification score of at least 12% in either ear (both relative to baseline). Prednisone could be started at screening but patients may have received prednisone prior to screening and the pre prednisone audiogram will be used as the screening audiogram for this study. It is expected the majority of these patients will screen for the study in this fashion as they are referred from otoloaryngology after initial treatment.

Able and willing to give written informed consent and comply with the requirements of the study protocol

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a loading dose of 320 mg of rilonacept followed by a once-weekly injection of 160 mg for 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Rilonacept

How Is the Trial Designed?

Group I: Rilonacept Experimental Treatment 1 Intervention

A loading dose of 320 mg the first dose then be a once-weekly injection of 160 mg for 24 weeks

Who Is Running the Clinical Trial?

Stanley Cohen

Lead Sponsor

Trials

1

Recruited

10+

Regeneron Pharmaceuticals

Industry Sponsor

Trials

690

Recruited

948,000+

Founded

1988

Headquarters

Tarrytown, USA

Known For

Precision medicine

Top Products

Dupixent, EYLEA, Libtayo, Praluent

Leonard Schleifer

Regeneron Pharmaceuticals

Chief Executive Officer since 1988

MD and PhD in Medicine

George Yancopoulos

Regeneron Pharmaceuticals

Chief Medical Officer since 1997

MD from Harvard Medical School

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12379981/

A Comprehensive Clinical Evaluation of Rilonacept in the ... In clinical studies, patients had an average of 2.6 previous recurrences, with an annualized recurrence rate (including initial occurrence) of ...

2. ahajournals.org ahajournals.org/doi/10.1161/JAHA.123.032516

Sustained Pericarditis Recurrence Risk Reduction With ... Rilonacept, a once‐weekly interleukin‐1 alpha and beta cytokine trap, reduced pericarditis recurrence in the phase 3 study, RHAPSODY.

3. arcalysthcp.com arcalysthcp.com/proven-efficacy

ARCALYST (rilonacept) RHAPSODY Clinical Trial Results In the RW period (primary efficacy end point)*: ARCALYST significantly reduced the risk of pericarditis recurrence. · 7% (2 of 30) of patients treated with ...

4. kiniksa.com kiniksa.com/ESC_2025_Poster_RESONANCE.pdf

Rilonacept Reduces Pericarditis Recurrence Risk recurrence rates before and after rilonacept initiation. Real world data from the RESONANCE patient registry demonstrate that rilonacept ...

5. aetna.com aetna.com/cpb/medical/data/700_799/0770.html

Rilonacept (Arcalyst) - Medical Clinical Policy Bulletins The authors concluded that this study presented 2-year safety and effectiveness data on rilonacept treatment in 5 patients with FCAS. The dramatic ...

6. arcalysthcp.com arcalysthcp.com/safety

ARCALYST (rilonacept) Side Effects & Safety In patients with DIRA, the most common adverse reactions (>10%) include upper respiratory tract infections, rash, otitis media, pharyngitis, and rhinorrhea.

7. mayoclinic.org mayoclinic.org/drugs-supplements/rilonacept-subcutaneous-route/description/drg-20071855

Rilonacept (subcutaneous route) - Side effects & dosage This medicine may cause a serious allergic reaction. Check with your doctor right away if you have a rash, itching, swelling of the face, tongue ...

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2008/125249lbl.pdf

Arcalyst (rilonacept) Label - accessdata.fda.gov The adverse events included injection site reactions and upper respiratory symptoms as were commonly seen in the adult subjects. The trough drug levels for four ...

9. drugs.com drugs.com/sfx/rilonacept-side-effects.html

Rilonacept Side Effects: Common, Severe, Long Term This medicine may cause a serious allergic reaction. Check with your doctor right away if you have a rash, itching, swelling of the face, tongue, and throat.

10. medlineplus.gov medlineplus.gov/druginfo/meds/a622030.html

Rilonacept Injection: MedlinePlus Drug Information injection site pain, bruising, bleeding, swelling, redness, warmth, or itching · nausea · diarrhea · upset stomach · stuffy nose · numbness · muscle ...