A Study Testing Safety and Efficacy of Transtympanic Paliroden for the Treatment of Cochlear Synaptopathy in Participants With Chronic, Constant Tinnitus (REACT)
A Randomized, Double-Blind, Placebo-Controlled, Single-Dose Phase 1b/2a Clinical Trial to Evaluate The Safety, Tolerability, Pharmacokinetics, and Efficacy of a Transtympanic Injection of Paliroden (CIL001) for the Treatment of Cochlear Synaptopathy in Participants With Chronic, Constant Tinnitus
The goal of this clinical trial is to investigate if paliroden (CIL001) is safe and can help treat cochlear synaptopathy in adult participants 18-75 years of age with normal hearing thresholds and normal cognition who exhibit chronic, constant tinnitus in both ears and have difficulty understanding speech in noisy environments. The main questions it aims to answer are:
- Is the study drug safe and tolerable over 6 months after injection into the middle ear?
- How much of the study drug is in the bloodstream at different time points after injection?
- How effective is a single dose of the study drug at treating cochlear synaptopathy in participants with chronic, constant tinnitus, as measured by hearing and listening tests?
Injection of the study drug will be compared to injection of a placebo (a substance with no study drug in it) to see if there is a difference.
Participants will be screened for eligibility and potentially receive a one-time injection into the middle ear of paliroden or placebo. The study lasts approximately 6 to 7 months if completing the whole study. Based on the visit windows, this could be between 190 and 218 days including a maximum of 42 days for Screening & Run-in and 175 days post-treatment.
Participants will:
- Visit the clinic for checkups and tests multiple times over the course of the study
- Complete a large battery of hearing and listening tests, answer questionnaires about their experiences, and provide blood samples for various tests
- Receive an injection of either the study drug or placebo (a substance that has no active drug in it), if eligible
調査の概要
研究の種類
入学 (推定)
段階
- フェーズ2
- フェーズ 1
参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
Inclusion Criteria:
- Native speaker of English
- Able and willing to give consent for the study
- Age 18-75 years (inclusive) at the time of signing the informed consent
- Report binaural, chronic, constant, subjective tinnitus for at least 6 months
- Hearing thresholds within normal limits (≤ 20 dB HL at 0.25 to 8 kHz, plus 3 and 6 kHz)
- Speech-in-noise deficit (at least 3 dB SNR loss in comparison to normative value of the AE-Matrix test) in both ears
- No concurrent tinnitus treatment
- Be considered as reliable and capable of adhering to the protocol, according to the judgment of the investigator
- Signed informed consent as described in § 11.3 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
Exclusion Criteria:
- Montreal Cognitive Assessment (MoCA) score < 26
- History of somatic/objective tinnitus as defined by AAO-HNS guidelines (e.g., pulsatile/whooshing sounds pulsating in synchrony with heartbeat, or caused by temporo-mandibular joint dysfunction)
- Known otologic pathology (e.g., otosclerosis, history of autoimmune hearing loss, radiation-induced hearing loss, fluctuating hearing, endolymphatic hydrops, Ménière's disease, vestibular schwannoma, history of sudden sensorineural hearing loss, Neurofibromatosis Type 2)
- History of otologic surgery (apart from tympanostomy tube insertion if more than one year prior to inclusion visit)
- Untreated hypothyroidism
- Use of prohibited concomitant therapies as described in § 6.10
- Current use or history of platinum-based chemotherapy
- Concurrent malignancies that require treatment
- Abnormal otoscopy as defined by less than 90% of the tympanic membrane visible (e.g., occluding cerumen, ear drum perforation)
- Current middle ear pathology (e.g., otitis media, tympanic membrane perforation)
- Abnormal tympanogram (other than type A)
- Conductive hearing loss (> 10 dB difference between air- and bone-conduction hearing thresholds)
- Asymmetric hearing thresholds (more than 15 dB difference between left and right ear) at 0.25 to 8 kHz, plus 3 and 6 kHz
- Lactation, known pregnancy, positive pregnancy test at either Screening (urine) or Baseline (serum) for Women of Childbearing Potential (WOCBP), or plan to become pregnant during the study
Liver Enzyme Lab Outcomes within the previous 2 months (or assessed at Baseline if not available at Screening ), see § 12.1.1:
- Bilirubin > upper limit normal (ULN)
- Liver transaminases (Aspartate Aminotransferase [AST] and Alanine Aminotransferase [ALT]) > 2 times ULN
- Gamma-glutamyltransferase (GGT) > 2 times ULN
Clinically significant electrocardiogram (ECG) abnormalities:
a. Marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval > 450 ms)
- History of additional risk factors for Torsades de Pointes (e.g., heart failure, hypokalemia, family history of Long QT Syndrome)
- Concurrent participation in another clinical study or participation in another clinical study within 30 days or five half-lives of the experimental drug (whichever is longer) prior to screening visit (V0)
- Diagnosed anxiety disorders, psychosis, depression, schizophrenia, attempted suicide, or other significant psychiatric conditions that could impact their ability to cooperate and comply with the study protocol
- Major surgery (at the discretion of the Principal Investigator or medically qualified designee) within eight weeks before screening or scheduled/planned surgery within the time frame of the study
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:トリプル
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的:paliroden (CIL001)
Those receiving a one-time unilateral transtympanic injection of 0.4mL of paliroden (CIL001)
|
One-time unilateral transtympanic injection of 0.4mL of paliroden (CIL001)
|
|
プラセボコンパレーター:Placebo
Those receiving a one-time unilateral transtympanic injection of 0.4mL of non-matching placebo
|
One-time unilateral transtympanic injection of 0.4mL of non-matching placebo
|
この研究は何を測定していますか?
主要な結果の測定
結果測定 |
時間枠 |
|---|---|
|
Incidence, nature, and severity of adverse events (AEs) and serious adverse events (SAEs) from baseline to 6 months (168 days). Safety data, including all observed AEs with a particular focus on ear and auditory symptomatology.
時間枠:From baseline to 6 months (168 days) after injection
|
From baseline to 6 months (168 days) after injection
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
プラズマ濃度-時間曲線下面積(AUC)を用いたパリロデンの血漿中濃度の測定
時間枠:注射日から28日間
|
注射日から28日間
|
|
|
ピーク血漿濃度(Cmax)を用いてパリロデンの血漿中濃度を測定する
時間枠:注射日から28日間
|
注射日から28日間
|
|
|
Determine the time to maximum plasma concentration (Tmax).
時間枠:From the day of injection to 28 days
|
From the day of injection to 28 days
|
|
|
Change from baseline in auditory function using speech-in-noise intelligibility as measured by the English (US) Matrix Test (AE-Matrix) and Time Compressed Words.
時間枠:From baseline to day 28, day 56, day 84, and day 168
|
For AE-Matrix: Speech level will be fixed at 65 dB HL, while noise level will be adjusted based on responses to determine levels at which participants obtained a speech reception threshold of 50% (in dB). For Time Compressed Words: This test consists of 50 carrier phrase and word stimuli from the Northwestern University Auditory Test No. 6 (NU-6) pool of 200 words that are compressed 60% (i.e., 60% of the carrier phrase and word has been removed). Participants will be asked to repeat the words they hear. Speech will be presented at 55 dB HL. Performance will be assessed by counting the number of words repeated correctly. |
From baseline to day 28, day 56, day 84, and day 168
|
協力者と研究者
スポンサー
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
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