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A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy

2026年10月27日 更新者：Grünenthal GmbH

A Randomized, Open-label Phase 2 Trial Consisting of a 24-Week Core Phase Followed by a 72-Week Extension Phase to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat Oral Solution in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy (DMD)

This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy (DMD). Participants will be randomized to one of three tegacorat treatment groups during a 24-week Core Phase. Participants who complete the Core Phase will continue into a 72-week Extension Phase. The study will assess muscle function, ambulatory performance, safety outcomes, pharmacokinetics, pharmacodynamic biomarkers, and health-related quality of life over a total treatment period of up to 96 weeks.

研究概览

地位

尚未招聘

条件

干预/治疗

详细说明

DMD is a severe, progressive neuromuscular disorder caused by mutations in the dystrophin gene, resulting in muscle degeneration and loss of function. Current standard-of-care glucocorticoid therapy may slow disease progression but is associated with significant long-term adverse effects. Tegacorat is a selective glucocorticoid receptor agonist and modulator (SEGRAM) that has demonstrated anti-inflammatory activity with reduced glucocorticoid receptor-mediated transactivation in clinical and nonclinical studies.

研究类型

介入性

注册 (估计的)

36

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。

学习联系方式

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

4年 至 6年 (孩子)

接受健康志愿者

不

有资格学习的性别

男性

描述

INCLUSION CRITERIA:

  • Male participant aged ≥4 years and <7 years at randomization.
  • Clinically and genetically confirmed diagnosis of Duchenne muscular dystrophy.
  • Body weight between 13 kg and 35 kg at screening.
  • Able to walk independently without assistive devices.
  • Able to complete the TTSTAND assessment without assistance in less than 10 seconds.
  • Evidence of varicella immunity by serology or vaccination history.
  • Parent(s)/legal guardian(s) provide informed consent and participant assent as required.

EXCLUSION CRITERIA:

  • Moderate or severe renal impairment.
  • Moderate or severe hepatic impairment.
  • Clinically significant abnormal laboratory findings.
  • Chronic or latent infection that may increase study risk.
  • Acute illness that could affect safety.
  • Symptomatic cardiomyopathy.
  • Diabetes mellitus.
  • Severe behavioral, cognitive, or psychiatric disorders interfering with study participation.
  • Adrenal insufficiency.
  • Clinically significant ECG abnormalities.
  • Hypersensitivity to study intervention or its components.
  • Current or recent use of prohibited treatments or investigational products.
  • Prior exposure to delandistrogene moxeparvovec.
  • Participation in another clinical trial within the protocol-defined washout period.

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：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：Tegacorat Low dose

Tegacorat Treatment Group 1
药品：Tegacorat

Oral solution
实验性的：Tegacorat Mid Dose

Tegacorat Treatment Group 2
药品：Tegacorat

Oral solution
实验性的：Tegacorat High dose

Tegacorat Treatment Group 3
药品：Tegacorat

Oral solution

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
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
大体时间：Baseline to Week 24

Change from baseline to Week 24 in TTSTAND velocity.
Baseline to Week 24
Extension Phase: Incidence of Adverse Events
大体时间：First dose through Week 96

Safety and Tolerability.
First dose through Week 96

次要结果测量

结果测量
措施说明
大体时间
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
大体时间：Baseline to Week 24

Change from baseline to Week 24 in SV95C measured using wearable sensor technology.
Baseline to Week 24
Core Phase: Change From Baseline in 10-Meter Walk/Run (10MWR) velocity
大体时间：Baseline to Week 24

Change From Baseline in Functional Test Performance.
Baseline to Week 24
Core Phase: Change From Baseline in Time-to-Climb-4-Stairs (TTCLIMB) velocity
大体时间：Baseline to Week 24

Change From Baseline in Functional Test Performance.
Baseline to Week 24
Core Phase: Incidence of Adverse Events
大体时间：First dose through Week 24

Safety and Tolerability.
First dose through Week 24
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
大体时间：Up to Week 96

Time to permanent discontinuation of study treatment due to an adverse event.
Up to Week 96
Extension Phase: Change From Baseline in Weight and BMI Z-Scores
大体时间：Baseline to Week 96

Change from baseline in weight and body mass index (BMI) Z-scores.
Baseline to Week 96
Extension Phase: Change From Baseline in Cushingoid Features
大体时间：Baseline to Week 96

Change from baseline in Cushingoid features.
Baseline to Week 96
Extension Phase: Change From Baseline in Glucose Metabolism
大体时间：Baseline to Week 96

Change from baseline in fasting glucose and fasting serum insulin.
Baseline to Week 96
Extension Phase: Change From Baseline in PARS-III Score
大体时间：Baseline to Week 96

Change from baseline in Personal Adjustment and Role Skills Scale (PARS-III) score.
Baseline to Week 96
Extension Phase: Change From Baseline in Bone Mineral Density
大体时间：Baseline to Week 96

Change from baseline in bone mineral density assessed by dual-energy X-ray absorptiometry (DXA).
Baseline to Week 96
Extension Phase: Vertebral Fracture Assessment
大体时间：Baseline to Week 96

Assessment of vertebral fractures by lateral thoracolumbar spine X-ray.
Baseline to Week 96
Extension Phase: Change From Baseline in Height Z-Score
大体时间：Baseline to Week 96

Change from baseline in height Z-score.
Baseline to Week 96
Extension Phase: Change From Baseline in Bone Age
大体时间：Baseline to Week 96

Change from baseline in bone age assessed by left hand X-ray.
Baseline to Week 96
Extension Phase: Change From Baseline in TTSTAND Velocity
大体时间：Baseline to Week 96

Change From Baseline in Functional Test Performance.
Baseline to Week 96
Extension Phase: Change From Baseline in 10MWR Velocity
大体时间：Baseline to Week 96

Change From Baseline in Functional Test Performance.
Baseline to Week 96
Extension Phase: Change From Baseline in TTCLIMB Velocity
大体时间：Baseline to Week 96

Change From Baseline in Functional Test Performance.
Baseline to Week 96
Extension Phase: Change From Baseline in SV95C
大体时间：Baseline to Week 96

Change from baseline in SV95C measured using wearable sensor technology.
Baseline to Week 96

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

Grünenthal GmbH

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 (估计的)

2026年11月9日

初级完成 (估计的)

2030年1月4日

研究完成 (估计的)

2030年1月4日

研究注册日期

首次提交

2026年10月27日

首先提交符合 QC 标准的

2026年10月27日

首次发布 (实际的)

2026年11月3日

研究记录更新

最后更新发布 (实际的)

2026年11月3日

上次提交的符合 QC 标准的更新

2026年10月27日

最后验证

2026年10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

  • KF6019-02
  • 2026-526326-40-00 (克蒂斯)
  • U1111-1339-2419 (其他标识符：Universal Trial Number)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

Information available on the Grünenthal Group Web Site (https://www.grunenthal.com/science/clinical-trial-transparency/data-sharing) and according to the European Federation of Pharmaceutical Industries and Associations (EFPIA) Data Sharing Principles

IPD 共享支持信息类型

  • 研究方案
  • 国际碳纤维联合会
  • 企业社会责任

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

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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