Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) (FORZETTO)
2026年5月20日 更新者：Dyne Therapeutics
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
研究概览
地位
招聘中
条件
详细说明
The study consists of three periods: a Screening period (up to 6 weeks), a Placebo-Controlled Period (72 weeks) and an open-label Long-Term Extension Period (96 weeks).
研究类型
介入性
注册 (估计的)
90
阶段
- 第三阶段
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
- 姓名：Dyne Clinical Trials
- 电话号码：+1-781-317-1919
- 邮箱：clinicaltrials@dyne-tx.com
学习地点
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North Carolina
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Hillsborough、North Carolina、美国、27278
- 招聘中
- Rare Disease Research, LLC
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接触：
- Hannah Nation
- 电话号码：984-314-2252
- 邮箱：hannah.nation@rarediseaseresearch.com
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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
- 孩子
- 成人
接受健康志愿者
不
描述
Inclusion Criteria:
- Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .
- Rise From Floor (RFF) time must be < 10 seconds for both screening assessments .
- Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)
Exclusion Criteria:
- Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization
- Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization
- Any change in prophylaxis/treatment for congestive heart failure (CHF) within 12 weeks prior to randomization
- Receipt of eteplirsen within 1 week prior to randomization
- Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization
- Receipt of givinostat within 12 weeks prior to randomization
- Receipt of gene therapy at any time
Note: Other inclusion or exclusion criteria may apply
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 |
|---|---|
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实验性的：Placebo-Controlled Period: Zeleciment Rostudirsen (DYNE-251)
Participants will be randomized to receive zeleciment rostudirsen, once every 4 weeks (Q4W) for up to 72 weeks.
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Administered by IV infusion
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安慰剂比较：Placebo-Controlled Period: Placebo
Participants will be randomized to receive placebo, Q4W for up to 72 weeks.
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通过静脉输液给药
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实验性的：Open-Label Long-Term Extension Period: Zeleciment Rostudirsen (DYNE-251)
All participants who complete the Placebo-Controlled Period of the study will receive zeleciment rostudirsen administered Q4W for up to 96 weeks.
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Administered by IV infusion
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研究衡量的是什么？
主要结果指标
结果测量 |
大体时间 |
|---|---|
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Rise From Floor (RFF) velocity
大体时间：Baseline, Week 73
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Baseline, Week 73
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次要结果测量
结果测量 |
大体时间 |
|---|---|
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RFF (Rise From Floor) velocity
大体时间：Baseline, up to Week 169
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Baseline, up to Week 169
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Stride Velocity 95th Percentile (SV95C)
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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North Star Ambulatory Assessment (NSAA) Total Score
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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10-Meter Walk/Run (10MWR) Velocity
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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4-Stair Climb (4SC) velocity
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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Functional Composite score
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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Forced Vital Capacity (FVC)
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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Patient Global Impression of Severity (PGI-S)
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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Outcome of Patient Global Impression of Change (PGI-C)
大体时间：Week 73, up to Week 169
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Week 73, up to Week 169
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Blood Creatine Kinase (CK) levels
大体时间：Baseline, Week 73, up to Week 169
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Baseline, Week 73, up to Week 169
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Incidence of participants With Treatment-Emergent Adverse Events (TEAEs)
大体时间：Through study completion, up to Week 173
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Through study completion, up to Week 173
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Maximum Observed Plasma Drug Concentration of DYNE-251 (Cmax)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Time to Maximum Observed Plasma Drug Concentration of DYNE-251 (tmax)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Area Under the Plasma Drug Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration of DYNE-251 in Plasma (AUC-tlast)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Area Under the Plasma Drug Concentration Versus Time Curve From Time 0 (Dosing) Extrapolated to Time Infinity of DYNE-251 (AUC∞)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Apparent Terminal Phase Elimination Rate Constant of DYNE-251 in Plasma (λz)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Apparent Terminal Elimination Half-Life of DYNE-251 in Plasma (t½)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Total Body Clearance (CL) of DYNE-251
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Volume of Distribution at the Terminal Phase of DYNE-251 in Plasma (Vz)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Volume of Distribution at Steady State of DYNE-251 in Plasma (Vss)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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Incidence of Participants With Antidrug Antibodies (ADAs)
大体时间：Through study completion, up to Week 169
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Through study completion, up to Week 169
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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。
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6月1日
初级完成 (估计的)
2030年12月1日
研究完成 (估计的)
2032年10月1日
研究注册日期
首次提交
2026年5月20日
首先提交符合 QC 标准的
2026年5月20日
首次发布 (实际的)
2026年5月27日
研究记录更新
最后更新发布 (实际的)
2026年5月27日
上次提交的符合 QC 标准的更新
2026年5月20日
最后验证
2026年5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
- DYNE251-DMD-301
- 2025-524096-23-00 (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
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.