- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT07826507
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
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Tipo de estudo
Inscrição (Estimado)
Estágio
- Fase 2
- Fase 1
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
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
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Triplo
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: 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)
|
|
Comparador de Placebo: 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
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
|---|---|
|
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.
Prazo: From baseline to 6 months (168 days) after injection
|
From baseline to 6 months (168 days) after injection
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Determinar a concentração no plasma de paliroden com Área sob a curva da concentração plasmática em função do tempo (AUC)
Prazo: Desde o dia da injeção até 28 dias
|
Desde o dia da injeção até 28 dias
|
|
|
Determinar a concentração no plasma de paliroden com Concentração Plasmática Máxima (Cmax)
Prazo: Desde o dia da injeção até 28 dias
|
Desde o dia da injeção até 28 dias
|
|
|
Determine the time to maximum plasma concentration (Tmax).
Prazo: 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.
Prazo: 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
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- REACT
Plano para dados de participantes individuais (IPD)
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