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First Clinical Study to Evaluate Safety, Tolerability & PK of DX243 in Healthy Volunteers and Patients With Hearing Loss (DX243-101)

2026年6月4日 更新者：Dendrogenix

Double-blind, Randomised, Placebo-controlled Phase 1/2a Study of Safety, Tolerability and PK of Single and Repeated SC Doses of DX243 in Healthy Volunteers, and 1-month Safety and Efficacy in Moderately Severe Age-related Hearing Loss

Phase 2a: Multiple Ascending Dose (MAD) in male and female patients with hearing loss:

The study will be conducted according to a randomised, placebo-controlled, double-blind design. A total of 24 patients, otherwise healthy, aged up to 75 years old, with mild to moderate hearing loss will be included. Two cohorts of 12 male or female patients (no ratio is required) will receive two different flat doses (low dose and high dose) ofDX243 or placebo for 29 days using SC administration. In each cohort of 12 patients, 4 will be randomised to placebo and 8 to DX243, so at the end of Phase 2a, 8 patients will have received placebo, 8 the low dose and 8 the high dose of DX243.

The primary objective is to evaluate the safety and tolerability of DX243 administered subcutaneously after repeated doses. The secondary objectives are to detect preliminary signal of efficacy, using speech in noise tests, tonal and vocal audiometry, as well as tinnitus and quality of life.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

This is a first-in-human, randomised, double-blind, placebo-controlled Phase 1/2a study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of DX243, a novel investigational compound, in healthy volunteers and patients with mild to moderate age-related hearing loss (ARHL). The study consists of two parts: a Phase 1 segment including single and multiple ascending dose evaluations in healthy volunteers, followed by a Phase 2a extension in patients with hearing impairment. The overall design allows for dose selection and regimen optimisation based on safety and PK results obtained in earlier cohorts.

In the Phase 2a part, 24 otherwise healthy male and female patients aged up to 75 years with up to moderately severe hearing loss will be enrolled. Patients will be randomised in a double-blind manner to receive either DX243 (two dose levels) or placebo. Treatment will consist of once-weekly subcutaneous administrations over 29 days (five injections), with 2 dosing levels selected based on Phase 1 safety, PK and modelling data to achieve target plasma concentrations while minimising local tolerability issues.

The primary objective of the Phase 2a portion is to assess the safety and tolerability of repeated subcutaneous administration of DX243, including treatment-emergent adverse events, local injection site reactions, laboratory parameters, and ECG findings. Secondary and exploratory objectives include characterisation of PK parameters and evaluation of preliminary efficacy signals using a comprehensive battery of audiological tests (including speech-in-noise performance, pure tone audiometry, and auditory brainstem response), tinnitus assessments, and quality-of-life measures. Patients will be followed for up to approximately 6 months after treatment to assess longer-term safety and durability of response.

研究类型

介入性

注册 (估计的)

24

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 比利时
    • Liège
      • Liège、Liège、比利时、4000
        • 招聘中
        • Centre hospitalier Universitaire de Liège Sart Tilman
        • 接触：

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

Inclusion Criteria:

  • Patient having self-reported recent difficulty hearing in noisy environments for at least 6 months prior to screening.
  • Patient exhibiting a speech-in-noise hearing deficit in at least one ear;
  • Patient having audiometrically-defined normal hearing or up to moderately severe hearing impairment

Exclusion Criteria:

  • Current tympanic membrane perforation;
  • Current acute or chronic otitis;
  • Genetic hearing loss;
  • Symmetric or asymmetric severe hearing loss;
  • Any therapy known as ototoxic;
  • Acute chronic otitis media or otitis externa terminated less than 7 days prior to randomisation;
  • History of chronic inflammatory or suppurative ear disease or cholesteatoma;
  • History of otosclerosis, suspected perilymph fistula or membrane rupture, suspected retro-cochlear lesion, barotrauma;
  • Prior ear surgery of any kind;
  • Fluctuating hearing loss;
  • Patient with conductive hearing loss;
  • History of Meniere's disease, autoimmune hearing loss, radiation-induced hearing loss, acoustic neuroma

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：DX243 low flat dose
Flat dose administered sub-cutaneously for 1 month
药品：DX243
DX243 10 mg/mL
实验性的：DX243 high flat dose
Flat dose administered sub-cutaneously for 1 month
药品：DX243
DX243 10 mg/mL
安慰剂比较：Placebo
Placebo administered sub-cutaneously for 1 month
药品：Placebo
Placebo solution, matching the external appearance of DX243.

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Safety and tolerability: incidence, nature, severity and causality of treatment emergent adverse events (TEAE) and (Serious) Adverse events (S)(AEs)
大体时间：Through study completion, an average 7 months
Through study completion, an average 7 months
Tolerability at the injection sites using a 1-5 scale Common Terminology Criteria for Adverse Events (CTCAE) for pain, erythema, swelling, induration, nodule and ulceration with 1 : normal and 5: worst possible symptom/sign
大体时间：For 14 days or up to normalisation after each administration whichever comes first
For 14 days or up to normalisation after each administration whichever comes first
Number of participants with at least one clinically significant abnormal laboratory result
大体时间：For 7 months

Prespecified hematology, coagulation, and clinical chemistry laboratory tests will be measured in standard local laboratory units throughout the study. Each analyte will be assessed individually against the applicable reference range and for clinical significance.

For this outcome measure, a patient will be counted once if he/she has at least one post-baseline abnormal clinically relevant laboratory value.
For 7 months
Number of Participants With Abnormal ECG Findings
大体时间：For 7 months

Twelve-lead triplicate ECGs will be obtained at each scheduled assessment throughout the study. PR interval, QRS duration, QT interval, and QTcF interval will be measured on each of the three ECG tracings. Overall ECG interpretation (normal/abnormal) and clinical significance of any abnormality will be determined by the investigator or qualified reader based on review of the triplicate ECG assessment.

For this outcome measure, a participant will be counted once if at least one post-baseline ECG assessment is abnormal.
For 7 months

次要结果测量

结果测量
措施说明
大体时间
Change in Pure Tone Audiometry
大体时间：For 7 months
For 7 months
Change in speech audiometry
大体时间：For 7 months
For 7 months
Hearing Handicap Inventory for the Elderly- Screening (HHIE-S) questionnaire score
大体时间：For 7 months
0 to 8 = 13% probability of hearing impairment (no handicap/no referal) 10 to 24= 50% probability of hearing impairment (mild-moderate handicap/refer) 26 to 40=84% probability of hearing impairment (severe handicap/refer)
For 7 months
Severity of tinnitus
大体时间：For 7 months
Tinnitus assessment (acouphenometry) and Tinnitus Functional Index (TFI)
For 7 months
Distorsion Product OtoAcoustic Emission (DPOAE)
大体时间：For 7 months
For 7 months
Quality of Life (QoL) assessed using 15iSSQ scale
大体时间：For 7 months
The 15-item Speech, Spatial, and Qualities of Hearing Scale is a 15-item questionnaire that evaluates hearing-related quality of life. It measures self-reported abilities in three specific hearing domains: Speech, Spatial, and Qualities of hearing. Higher scores indicate better hearing performance and fewer limitations in daily life.
For 7 months

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Dendrogenix

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2026年5月13日

初级完成 (估计的)

2027年10月1日

研究完成 (估计的)

2027年10月1日

研究注册日期

首次提交

2026年4月30日

首先提交符合 QC 标准的

2026年6月4日

首次发布 (实际的)

2026年6月10日

研究记录更新

最后更新发布 (实际的)

2026年6月10日

上次提交的符合 QC 标准的更新

2026年6月4日

最后验证

2026年6月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • DX243-101
  • 2023-505778-14-00 (克蒂斯)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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