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
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Tipo di studio
Tipo di studio
Iscrizione (Stimato)
Iscrizione
Fase
Fase
- Fase 2
- Fase 1
Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Triplicare
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
|---|---|
|
Sperimentale: paliroden (CIL001)
Those receiving a one-time unilateral transtympanic injection of 0.4mL of paliroden (CIL001)
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One-time unilateral transtympanic injection of 0.4mL of paliroden (CIL001)
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Comparatore placebo: Placebo
Those receiving a one-time unilateral transtympanic injection of 0.4mL of non-matching placebo
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One-time unilateral transtympanic injection of 0.4mL of non-matching placebo
|
Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Lasso di tempo |
|---|---|
|
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.
Lasso di tempo: From baseline to 6 months (168 days) after injection
|
From baseline to 6 months (168 days) after injection
|
Misure di risultato secondarie
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Determinare la concentrazione plasmatica di paliroden con l'Area sotto la curva della concentrazione plasmatica in funzione del tempo (AUC)
Lasso di tempo: Dal giorno dell'iniezione fino a 28 giorni
|
Dal giorno dell'iniezione fino a 28 giorni
|
|
|
Determinare la concentrazione plasmatica di paliroden con la Concentrazione Plasmatica Massima (Cmax)
Lasso di tempo: Dal giorno dell'iniezione a 28 giorni
|
Dal giorno dell'iniezione a 28 giorni
|
|
|
Determine the time to maximum plasma concentration (Tmax).
Lasso di tempo: 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.
Lasso di tempo: 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
|
Collaboratori e investigatori
Sponsor
Sponsor
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Inizio studio
Completamento primario (Stimato)
Completamento primario
Completamento dello studio (Stimato)
Completamento dello studio
Date di iscrizione allo studio
Primo inviato
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Primo Inserito
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento pubblicato
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
Altri numeri di identificazione dello studio
- REACT
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