2025-525124-10-00招募中2 期
A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
Entrada Therapeutics Inc.11 个研究点 分布在 4 个国家目标入组 56 人开始时间: 2026年8月26日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 56
- 试验地点
- 11
- 主要终点
- Incidence and severity of treatment emergent adverse events (TEAEs).
研究概览
简要总结
To evaluate the long-term safety and tolerability of study drug in participants with Duchenne muscular dystrophy (DMD).
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Willing and able to provide consent (if at age of majority) or assent (if a minor).
- •Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-
- •Males who are sexually active with a female partner of childbearing potential must agree to use condoms during sexual intercourse.
排除标准
- •Any change from the applicable parent study eligibility criteria, including safety events during the parent study, that in the opinion of the investigator in consultation with the medical monitor and/or sponsor designee precludes safe use of study drug.
研究组 & 干预措施
ENTR-601-45
Test
干预措施: ENTR-601-45 (Drug)
ENTR-601-44
Test
干预措施: ENTR-601-44 (Drug)
结局指标
主要结局
Incidence and severity of treatment emergent adverse events (TEAEs).
Incidence and severity of treatment emergent adverse events (TEAEs).
Changes in vital sign measurements.
Changes in vital sign measurements.
Changes in clinical laboratory results.
Changes in clinical laboratory results.
Changes in electrocardiogram (ECG) parameters.
Changes in electrocardiogram (ECG) parameters.
Changes in physical examination findings.
Changes in physical examination findings.
次要结局
- Anti-drug antibody (ADA) and anti-dystrophin antibody in serum.
- Plasma concentration of study drug compounds and their final metabolite.
- Change from parent study Part A and OL Period baselines to LTE EOS in 10-Meter Walk/Run (10MWR).
- Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF).
- Change from parent study Part A and OL Period baselines to LTE EOS in Timed 4-Stair Climb (4SC).
- Change from parent study Part A and OL Period baselines to LTE EOS in stride velocity 95th centile (SV95C).
- Change from parent study Part A and OL Period baselines to LTE EOS in North Star Ambulatory Assessment (NSAA).
- Change from parent study Part A and OL Period baselines to LTE EOS in Performance of the Upper Limb v2.0 (PUL 2.0).
研究者
Regulatory Affairs
Scientific
Entrada Therapeutics Inc.
研究点 (11)
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