NCT05096221已完成3 期
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 126
- 试验地点
- 82
- 主要终点
- Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
研究概览
简要总结
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 7 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Is ambulatory and from 4 to under 8 years of age at time of randomization.
- •Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
- •Ability to cooperate with motor assessment testing.
- •Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
- •rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
- •A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45.
排除标准
- •Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
- •Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- •Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.
- •Other inclusion or exclusion criteria could apply.
结局指标
主要结局
Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
时间窗: Baseline, Week 52 (Part 1)
The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total score is defined as the sum of all 17 items, ranging from 0 (worst) to 34 (best). The response vector consists of the change from baseline in NSAA total score at the post-baseline visit. The model includes the covariates of treatment group, visit, treatment group by visit interaction, age group, baseline NSAA total score, and baseline NSAA total score by visit interaction. All covariates are fixed effects in this analysis. An increase in score indicates an improvement in motor function.
次要结局
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content(Week 12)
- Part 1: Change From Baseline in Time to Rise From the Floor at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in the Timed Stair Ascend 4 Steps Test at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in PROMIS Score in Upper Extremity Function to Week 52(Baseline, Week 52 (Part 1))
- Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA(Week 52 (Part 1))
- Parts 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)(Up to 104 weeks)
- Parts 1 and 2: Number of Participants With Adverse Events of Special Interest (AESI)(Up to 104 weeks)
- Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content(Week 12)
- Part 1: Change From Baseline in Time to Rise From the Floor at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Time to Complete 100 Meter Walk/Run (100MWR) at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in the Timed Stair Ascend 4 Steps Test at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Stride Velocity 95th Centile (SV95C) at Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Score in Mobility to Week 52(Baseline, Week 52 (Part 1))
- Part 1: Change From Baseline in PROMIS Score in Upper Extremity Function to Week 52(Baseline, Week 52 (Part 1))
- Part 1: Number of Skills Gained or Improved at Week 52 as Measured by the NSAA(Week 52 (Part 1))
研究者
研究点 (82)
Loading locations...
相似试验
进行中（未招募）
3 期
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular DystrophyDuchenne Muscular DystrophyNCT04281485Pfizer114
进行中（未招募）
3 期
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyNCT05881408Sarepta Therapeutics, Inc.148
已完成
1 期
A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)Muscular Dystrophy, DuchenneNCT03769116Sarepta Therapeutics, Inc.41
终止
2 期
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneNCT05429372Pfizer10
终止
3 期
Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMAMDSRefractory Anemia With Excess BlastsRAEBMyelodysplastic SyndromeNCT02562443Traws Pharma, Inc.372
相关资讯
Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant.
• uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy.
• Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.last yearElevidys Gene Therapy for Duchenne Shows Sustained Benefit in Long-Term Follow-Up- Five-year follow-up data from Study 101 supports the role of delandistrogene moxeparvovec (Elevidys) in stabilizing or slowing Duchenne muscular dystrophy (DMD) progression.
- Patients treated with Elevidys maintained 10-meter walk/run time over five years, showing a clinically meaningful difference compared to the external control cohort.
- EMBARK trial data indicates Elevidys does not negatively impact cardiac measures over a 52-week treatment period, reinforcing its manageable safety profile.
- A pre-specified global statistical test in the EMBARK trial indicated a functional treatment effect, despite not meeting the primary endpoint of change in NSAA score.last year