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)
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 80
- 试验地点
- 17
- 主要终点
- Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period)
研究概览
简要总结
This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).
The investigational medicines are currently being investigated in multiple ascending dose parent studies. After participants complete their respective parent study, there is a need to understand the effects of long-term administration of ENTR-601-44 and ENTR-601-45.
Participants enrolling in this study will begin this long-term extension (LTE) study at the dose level they received upon completion of the parent study with possible dose escalation in the LTE study based on emerging safety and efficacy data from the parent studies.
Participants will:
- Receive study treatment in the form of multiple intravenous (IV) infusions (slow injections) into a vein over the course of several weeks
- Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and excersice tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a results of the study drug.
Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 20 Years（Child, Adult）
- 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-601-45-201
- •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
- •Participant has a condition or circumstance that in the view of the investigator places the subject at high risk of poor treatment compliance or for not completing the study
研究组 & 干预措施
ENTR-601-45
ENTR-601-45 intravenous infusion every 6 weeks
干预措施: ENTR-601-45 (Drug)
ENTR-601-44
ENTR-601-44 intravenous infusion every 6 weeks
干预措施: ENTR-601-44 (Drug)
结局指标
主要结局
Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period)
时间窗: From baseline through End of Study (up to 2 years).
Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.
次要结局
- Plasma concentration of study drug compounds and their final metabolite(From baseline through End of Study (up to 2 years).)
- Change from parent study Part A and OL Period baselines to LTE EOS in 10-Meter Walk/Run (10MWR)(From baseline through End of Study (up to 2 years).)
- Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF)(From baseline through End of Study (up to 2 years).)
- Change from parent study Part A and OL Period baselines to LTE EOS in Timed 4-Stair Climb (4SC)(From baseline through End of Study (up to 2 years).)
- Change from parent study Part A and OL Period baselines to LTE EOS in stride velocity 95th centile (SV95C)(From baseline through End of Study (up to 2 years).)
- Change from parent study Part A and OL Period baselines to LTE EOS in North Star Ambulatory Assessment (NSAA)(From baseline through End of Study (up to 2 years).)
- Change from parent study Part A and OL Period baselines to LTE EOS in Performance of the Upper Limb v2.0 (PUL 2.0)(From baseline through End of Study (up to 2 years).)
- Anti-drug antibody (ADA) and anti-dystrophin antibody in serum(From baseline through End of Study (up to 2 years).)