Using atorvastatin to prevent hearing loss in head and neck cancer patients receiving cisplatin

Phase II Interventional Study Using Atorvastatin to Reduce Cisplatin-Induced Hearing Loss Among Individuals With Head and Neck Cancer

Phase 2 Interventional National Institutes of Health Clinical Center (CC) · NCT04915183

This study is testing if atorvastatin can help prevent hearing loss in adults with head and neck cancer who are getting chemotherapy with cisplatin.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment224 (estimated)
Ages18 Years to 100 Years
SexAll
SponsorNational Institutes of Health Clinical Center (CC) NIH
Drugs / interventionschemotherapy, immunotherapy, radiation
Locations5 sites (Atlanta, Georgia and 4 other locations)
Trial IDNCT04915183 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of atorvastatin, a cholesterol-lowering medication, in reducing hearing loss among individuals with head and neck squamous cell carcinoma undergoing cisplatin-based chemotherapy and radiation. Participants aged 18 and older will be divided into two groups: one receiving atorvastatin and the other receiving a placebo. The study will evaluate changes in hearing sensitivity before and after treatment, with assessments conducted at three different time points. The primary goal is to determine if atorvastatin can significantly reduce the incidence of hearing loss compared to placebo.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older diagnosed with squamous cell carcinoma of the head and neck who will receive cisplatin-based chemotherapy and radiation.

Not a fit: Patients who are not undergoing cisplatin treatment or those with other types of cancer may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could help preserve hearing in cancer patients undergoing cisplatin treatment, improving their quality of life.

How similar studies have performed: While the use of atorvastatin for this specific purpose is novel, preclinical data suggest potential benefits, and similar approaches have shown promise in other contexts.

Eligibility criteria

Show full inclusion / exclusion criteria
* INCLUSION CRITERIA:

In order to be eligible to participate in this study, an individual must meet all of the following criteria evaluated by the study team, including an on-site oncologist:

* Willingness and ability to comply with and participate in all study procedures and availability for the duration of the study
* Ability to provide consent and provision of signed and dated informed consent form
* Adult subjects, aged \>=18
* Diagnosed with squamous cell carcinoma of the head and neck, confirmed by a pathologic review of surgical or biopsy specimen(s), who meet standard clinical and laboratory criteria and will undergo treatment with concomitant cisplatin-based chemotherapy and radiation with curative intent or treatment with neoadjuvant chemotherapy (NAC). This includes subjects who will be treated with either intensity-modulated radiation therapy (IMRT) or proton radiotherapy, with planned dose to the cochlea \<35 Gy (to limit confounding effects of radiation). Patients receiving immunotherapy in addition to cisplatin chemoradiation are eligible. Subjects treated with either high-dose cisplatin (typically 100 mg/m\^2 x 2-3 doses every three weeks) or low-dose, weekly cisplatin (typically 40 mg/m\^2 x 6-7 doses weekly) may enroll. Cisplatin in the neo-adjuvant setting may be given within a combination chemotherapy regimen (e.g TPF= Docetaxel, Cisplatin and 5FU or DC = Docetaxel Cisplatin)
* Subjects must have hearing thresholds at or better than 70 dB HL at 1, 2, and 4 kHz in at least one ear at the time of their baseline audiogram. CTCAE criteria (primary endpoint) and ASHA criteria (secondary endpoint) are based on changes in hearing in at

least one ear with one ototoxicity event/grade assigned per person.

* Baseline laboratory tests with lab values \<1.5x the upper limit of normal: aspartate aminotransferase (AST or SGOT); alanine aminotransferase; creatine phosphokinase, creatinine
* Ability to take oral medication by mouth or by feeding tube and willingness to adhere to the daily atorvastatin or placebo regimen
* For females of reproductive potential: use of highly effective contraception for at least 1 month prior to enrollment and agreement to use such a method during study participation and for an additional 8 weeks after the end of atorvastatin administration

EXCLUSION CRITERIA:

An individual who meets any of the following criteria will be excluded from participation in this study:

* Subjects currently taking a statin drug
* Subjects with bilateral flat, Type B tympanogram
* Subjects with bilateral cochlear implants
* Pre-existing liver or kidney disease that in the opinion of the investigator would interfere with taking Atorvastatin 40 mg daily.
* Subjects with a history of prior treatment with platinum chemotherapy drugs
* Staff members of the NIDCD Sections and of the lead site investigators headed by the PIs
* Children will be excluded because HNSCC in children under age 18 is exceedingly rare
* Current use of cimetidine, spironolactone, ketoconazole, cyclosporine, protease inhibitors, gemfibrozil, clarithromycin or itraconazole
* Pregnancy, lactation, or plan to become pregnant
* Known allergic reactions to components of atorvastatin or the placebo
* Other severe or unstable medical conditions which clinical site PI believes increase risk to safety or ability to complete study
* Expected concomitant use of aminoglycoside antibiotics

Where this trial is running

Atlanta, Georgia and 4 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Hearing LossHead and Neck CancerAudiogramStatinChemotherapySquamous Cell Carcinoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.