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Study to Evaluate Safety and Efficacy of DB-020 to Protect Hearing in Patients Receiving Cisplatin for Cancer Treatment

Phase 1
Completed
Conditions
Ototoxicity
Interventions
Drug: Placebo
Registration Number
NCT04262336
Lead Sponsor
Decibel Therapeutics
Brief Summary

The purpose of this study is to evaluate whether DB-020 administered via an injection in the middle ear prevents hearing loss in participants who will receive high doses of cisplatin as part of their treatment for cancer.

Detailed Description

Cisplatin is a widely used and effective chemotherapy in the treatment of adult and pediatric solid tumors, including bladder, testicular, head and neck, and lung cancers. Serious side effects of cisplatin treatment include ototoxicity. To date, no approved therapy to prevent or treat ototoxicity exists for people receiving cisplatin treatment, which remains the most common dose-limiting side effect associated with cisplatin administration.

Participants who will be undergoing cancer treatment with high doses of cisplatin every 21 or 28 days will receive IT injection with one ear receiving DB-020 and one ear receiving placebo during each administration. The study will comprise 2 parts. In Part A, eligible participants will be randomized to one of two doses of DB-020. In Part B, participants will be treated with a single dose of DB-020, as selected from data collected in Part A. In both Parts A and B, the ear receiving DB-020 or placebo will be randomized. The choice of dose in Part B will depend on the data from Part A. If appropriate safety and efficacy is observed at either dose level, the Sponsor has the option of dosing at one of these concentrations either unilaterally (ie, with placebo administered in the contralateral ear) or bilaterally (ie, open-label DB-020 administered to both ears) in Part B.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
22
Inclusion Criteria
  • Ability to communicate with medical team and staff, willingness to participate in the study, give written informed consent, comply with the study restrictions
  • Adults aged 18 years, inclusive, or older
  • Treatment for cancer with Intervenous cisplatin once every 21 or 28 days
  • Plan to receive a minimum cumulative dose of cisplatin of ≥ 280 mg/m2 over at least three cycles
  • Concomitant use of other chemotherapy and radiation is permitted except investigational agents and/or radiation > 35 Grays involving the cochlear area
  • Male patients, their female partner(s), and female patients of childbearing potential must agree to use 2 forms of contraception, 1 of which must be a barrier method, during the study and for 90 days after the last study drug administration.
  • Male and female patients who consider themselves abstinent, and who agree to remain abstinent during the study and for 90 days after the last study drug administration
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
  • Anticipated survival > 1 year
  • Normal or not clinically significant otoscopic findings in both ears
  • Patient has read, understood, and voluntarily signed the informed consent form.
Exclusion Criteria
  • Female or male patients with female partners who are pregnant, lactating, or planning to attempt to become pregnant during this study or within 90 days after last dose of study drug
  • Prior treatment with a cisplatin regimen
  • Signs of disturbed integrity of the tympanic membrane on otoscopy or tympanometry
  • History of congenital hearing loss
  • History of otological surgery (excluding myringotomy tubes or simple tympanoplasty)
  • History of sudden hearing loss
  • History of conductive hearing loss > 10 decibels at 2 frequencies in either ear
  • Diagnosis of Meniere's disease
  • Diagnosis of autoimmune middle ear disease
  • Hearing loss greater than (not including) 45 decibels Hearing Loss averaged at 6 and 8 kilohertz in either ear
  • Asymmetry in hearing thresholds between left and right ear equal to or exceeding 20 decibels at any single frequency or 10 decibels at any 3 consecutive frequencies, up to and including 8 kilohertz
  • Previous radiation exposure > 35 Grays to all or part of the cochlea
  • Consumption of > 6 grams of salicylate or > 5 grams of acetaminophen (paracetamol) per day for the past month, or aminoglycoside use in the past month
  • Use of any investigational drug or device within 30 days prior to the first dose of study medication (6 months for biologic therapies) or 5 half-lives of the investigational drug, if known, whichever time is longer
  • History of any significant drug allergy (such as anaphylaxis or hepatotoxicity) and/or allergy to the excipients of the study medications
  • Presence of hepatitis C antibody with reflex hepatitis C virus (HCV) RNA testing (if anti-HCV is positive), hepatitis B surface antigen, or HIV antibodies 1 and 2

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
DB-020 for Injection, 12%/placeboDB-020dosage
DB-020 for Injection, 12%/placeboPlacebodosage
DB-020 for Injection, 25%/placeboPlacebodosage
DB-020 for Injection, 25%/placeboDB-020dosage
Primary Outcome Measures
NameTimeMethod
Number of patients with treatment-emergent Adverse Events (TEAEs) and/or abnormal changes from baseline in clinical laboratory abnormalities and/or vital signs and/or ECG assessmentsFrom screening/baseline (Day -28 to Day -2) or day of first dose of DB-020 (Cycle 1 Day 1) for up to 6 cycles (21 or 28 day cycles) through End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

To investigate the safety and tolerability of DB-020 when given intratympanically to patients receiving cisplatin chemotherapy treatment

Secondary Outcome Measures
NameTimeMethod
Changes from Baseline in Tinnitus Functional Index (TFI) Total Score compared to End of Treatment [Changes in Hearing]Baseline (Day -5 to Day -1) for up to 6 cycles (21 or 28 day cycles) through End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

The TFI is a 25-item self-assessment scale comprised of eight subscales (intrusiveness, sense of control, cognitive, sleep, auditory, relaxation, quality of life, and emotional) measuring the impact of tinnitus. Items are scored on a range of 0 to 10. The TFI Total Score is calculated as the mean of the 25 individual item scores multiplied by 10. The Total Score can range from 0 to 100. Higher scores indicate a greater degree of tinnitus-related impairment. Change from baseline values will be calculated as the reported TFI value minus the baseline value. A positive change from baseline indicates more tinnitus-related impairment

Changes from Baseline in Distortion Product Otoacoustic Emission (DPOAE) Values compared to End of Treatment [Changes in Hearing]Baseline (Day -5 to Day -1) and Cycle 1 Day 1 (21 or 28 day cycles) and End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

Distortion product otoacoustic emission (DPOAE) is defined as sound generated within the cochlea by stimulating the ear with two simultaneous tones of different frequency. DPOAEs serve as an objective measure of hearing sensitivity. Tones will be played from low to high frequencies (1 to 4 kHz) at soft to moderate levels to assess responses at different regions of the inner ear. DP levels will be recorded for each frequency and ear. Higher DP levels indicate more sensitive hearing. Change from baseline values will be calculated as the reported DP level value minus the baseline value. A negative change from baseline indicates less sensitive hearing

Area under the plasma concentration-time curve (AUC 0-inf) of free (unbound) cisplatin0.25 hours predose, mid-point of IV infusion, end of infusion, 0.25, 0.5, 1 & 2 hours postdose on Day 1 of each cycle (21 or 28 day cycles)
Incidence of Ototoxicity measured by American Speech-Language-Hearing Association (ASHA) criteriaBaseline (Day -5 to Day -1) for up to 6 cycles (21 or 28 day cycles) through End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

Air conduction audiometry consists of a set of pure tones presented to the patient through small speakers in a headset, played at carrier frequencies ranging from 250 to 16,000 Hz. Pure tone thresholds are recorded for each frequency tested. Ototoxicity will be defined according to the American Speech-Language-Hearing Association (ASHA) criteria for significant ototoxic change. Significant ototoxic change must meet one of the following three criteria: (i) ≥20 dB decrease at any one test frequency, (ii) ≥10 dB decrease at any two adjacent frequencies, or (iii) loss of response at three consecutive frequencies where responses were previously obtained. The presence of ototoxicity will be calculated for each ear

Changes from Baseline in Pure Tone Threshold Values compared to End of Treatment [Changes in Hearing]Baseline (Day -5 to Day -1) for up to 6 cycles (21 or 28 day cycles) through End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

Air conduction audiometry consists of a set of pure tones presented to the patient through small speakers in a headset, played at carrier frequencies ranging from 250 to 16,000 Hz. Pure tone thresholds are recorded for each frequency tested. Higher values indicate a greater degree of hearing impairment. Changes from baseline values will be calculated for each frequency and ear as the reported pure tone threshold value minus the baseline value. A negative change from baseline indicates hearing loss

Changes from Baseline in Words-in-Noise (WIN) Values compared to End of Treatment [Changes in Hearing]Baseline (Day -5 to Day -1) and Cycle 1 Day 1 (21 or 28 day cycles) and End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

The WIN test is a series of trials to measure the ability to accurately recognize speech in noise. In each trial, a series of 5 words is read to the listener with the instruction to repeat each word. The number of correct responses is captured. Trials are presented at 7 signal-to-noise ratios, from 0 to 24 dB, in steps of 4 dB. WIN thresholds are then calculated for each ear using the Spearman-Kaerber method. Higher WIN thresholds indicate better word recognition. Change from baseline values will be calculated for each ear as the reported WIN threshold value minus the baseline value. A negative change from baseline indicates worse word recognition

Time to reach maximum observed plasma concentration (tmax) of free (unbound) cisplatin0.25 hours predose, mid-point of IV infusion, end of infusion, 0.25, 0.5, 1 & 2 hours postdose on Day 1 of each cycle (21 or 28 day cycles)
Changes from Baseline in Hearing Handicap Inventory for Adults (HHIA) Total Score compared to End of Treatment [Changes in Hearing]Baseline (Day -5 to Day -1) and Cycle 1 Day 1 (21 or 28 day cycles) and End of Treatment Visit (28 days after last dose of study drug), up to 196 Days after first dose of study drug

The Hearing Handicap Inventory for Adults (HHIA) is a 25-item self-assessment scale comprised of two subscales (emotional and social/situational) measuring the impact of hearing loss. Items are scored on a range of 0 to 4. The HHIA Total Score is calculated as the sum of the 25 individual item scores. The HHIA Total Score can range from 0 to 100. Higher scores indicate a greater degree of hearing impairment. Change from baseline values will be calculated as the reported HHIA value minus the baseline value. A positive change from baseline indicates more hearing impairment

Plasma Concentrations of DB-020Predose and 0.25 hours prior to cisplatin administration on Cycle 1 Day 1 (21 or 28 day cycles)
Maximum observed plasma concentration (Cmax) of free (unbound) cisplatin0.25 hours predose, mid-point of IV infusion, end of infusion, 0.25, 0.5, 1 & 2 hours postdose on Day 1 of each cycle (21 or 28 day cycles)
Half-life (t1/2) of plasma concentrations of free (unbound) cisplatin0.25 hours predose, mid-point of IV infusion, end of infusion, 0.25, 0.5, 1 & 2 hours postdose on Day 1 of each cycle (21 or 28 day cycles)

Trial Locations

Locations (8)

Northwell Health Cancer Center

🇺🇸

Lake Success, New York, United States

Oncology and Palliative Care Research

🇦🇺

Melbourne, Victoria, Australia

WVU Cancer Institute

🇺🇸

Morgantown, West Virginia, United States

Queensland Head and Neck Cancer Centre

🇦🇺

Woolloongabba, Queensland, Australia

Peter MacCallum Cancer Centre

🇦🇺

Melbourne, Victoria, Australia

University of Miami Health System

🇺🇸

Miami, Florida, United States

Fiona Stanley Hospital, Clinical Trials Unit Cancer Center

🇦🇺

Murdoch, Western Australia, Australia

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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