NCT06246513进行中（未招募）3 期
A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 17
- 试验地点
- 15
- 主要终点
- Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
研究概览
简要总结
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 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 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cohort 1, only ambulatory participants:
- •Able to walk without assistive aid
- •10MWR <30 seconds
- •Cohort 2, only non-ambulatory participants:
- •10MWR ≥30 seconds or unable to perform
- •PUL 2.0 entry scale score ≥3
- •Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
- •Able to cooperate with muscle testing
- •Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.
排除标准
- •Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
- •Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
- •Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
- •Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)
- •Other inclusion/exclusion criteria apply.
研究组 & 干预措施
SRP-9003
Experimental
Participants will receive a single intravenous (IV) infusion of SRP-9003.
干预措施: SRP-9003 (Biological)
SRP-9003
Experimental
Participants will receive a single intravenous (IV) infusion of SRP-9003.
干预措施: Glucocorticoid (Drug)
结局指标
主要结局
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
时间窗: Baseline, Day 60
次要结局
- Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score(Baseline through Month 60)
- Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test(Baseline through Month 60)
- Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test(Baseline through Month 60)
- Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test(Baseline through Month 60)
- Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test(Baseline through Month 60)
- Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test(Baseline through Month 60)
- Time to Change of Loss of Ambulation(Baseline through Month 60)
- Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)(Baseline, Day 60)
- Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)(Baseline, Day 60)
- Change From Baseline Through Month 60 in Creatine Kinase Level(Baseline through Month 60)
- Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay(Baseline, Day 60)
- Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score(Baseline through Month 60)
- Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)(Baseline through Month 60)
研究者
研究点 (15)
Loading locations...
相似试验
进行中（未招募）
1 期
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)Limb Girdle Muscular DystrophyNCT05876780Sarepta Therapeutics, Inc.6
已完成
3 期
Acalabrutinib Safety Study in Untreated and Relapsed or Refractory Chronic Lymphocytic Leukemia PatientsChronic Lymphocytic LeukemiaNCT04008706AstraZeneca552
已完成
3 期
A Study on Safety and Efficacy of Tocilizumab (RoActemra/Actemra) Alone or in Combination With Non-Biologic Antirheumatics in Participants With Rheumatoid ArthritisRheumatoid ArthritisNCT01987479Hoffmann-La Roche150
进行中（未招募）
3 期
Study of Durvalumab and Tremelimumab as First-line Treatment in Patients With Advanced Hepatocellular CarcinomaHepatocellular CarcinomaNCT03298451AstraZeneca1,324
进行中（未招募）
3 期
A Study Evaluating the Efficacy and Safety of Adjuvant Giredestrant Compared With Physician's Choice of Adjuvant Endocrine Monotherapy in Participants With Estrogen Receptor-Positive, HER2-Negative Early Breast Cancer (lidERA Breast Cancer)Early Breast CancerNCT04961996Hoffmann-La Roche4,170