NCT06952686撤回3 期
A Seamless Phase 1/3, Multicenter, Single Dose Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Subjects (COMPASS)
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that will assess safety and efficacy.
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
- •Ambulatory, defined as able to walk without assistive aid, 10MWR <30 seconds, and NSAD total score ≥20
- •Non-ambulatory, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥3
- •Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic gamma-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to pre-Infusion screening. Results to be confirmed by sponsor at a CLIA/CAP/ISO15189 certified laboratory prior to dosing.
- •Have AAVrh74 antibody titers <1:400 (that is, not elevated) as determined by an enzyme-linked immunoassay
排除标准
- •Has a symptomatic infection (for example, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks before study treatment infusion
- •Part A: Has left ventricular ejection fraction (LVEF) <50% on the screening echocardiogram (ECHO) (without use of cardiac medication) or clinical signs and/or symptoms of cardiomyopathy or any history of cardiac disease
- •Part B: Has LVEF <40% on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy
- •Has FVC <40% of predicted value at screening and/or requirement for nocturnal ventilation
- •Serological evidence of current, chronic, or active human immunodeficiency virus infection, or hepatitis B or C infection or active viral or bacterial infection based on clinical observations
- •Any prior treatment with gene therapy, cell-based therapy (for example, stem cell transplantation), clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9), or any other form of gene editing
- •Treatment with human growth factor within 3 months of Day 1
- •Treatment with any investigational medication within 6 months of the screening visit
- •Is unable to undergo or tolerate a cardiac MRI procedure for any reason
- •Note: Other inclusion/exclusion criteria may apply.
研究组 & 干预措施
SRP-9005
Experimental
Participants will receive a single intravenous (IV) infusion of SRP-9005.
干预措施: Corticosteroid (Drug)
SRP-9005
Experimental
Participants will receive a single intravenous (IV) infusion of SRP-9005.
干预措施: SRP-9005 (Biological)
结局指标
主要结局
Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs)
时间窗: Baseline through Month 60
Part A and B: Change from Baseline in gamma-Sarcoglycan (SG) Expression at Day 60 Post-dose in Ambulatory Participants as Measured by Immunofluorescence (IF) Percent Positive Fibers (PPF)
时间窗: Baseline, Day 60
次要结局
- Part A and B: Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score(Baseline through Month 60)
- Part A and B: Change from Baseline through Month 60 in Time to Rise From Floor(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Time to Ascend 4 Steps(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Time to Complete the 10-meter Walk/Run (10MWR)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Timed Up and Go(Baseline through Month 60)
- Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by IF Percent Fiber Intensity (PFI)(Baseline, Day 60)
- Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by Western Assay(Baseline, Day 60)
- Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PPF(Baseline to Day 60)
- Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PFI(Baseline to Day 60)
- Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by Western Assay(Baseline to Day 60)
- Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PPF(Baseline to Month 24)
- Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PFI(Baseline to Month 24)
- Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by Western Assay(Baseline to Month 24)
- Part A and B: Change from Baseline Through Month 60 in Creatine Kinase Levels(Baseline through Month 60)
- Part A and B: Loss of Ambulation(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imaging (MRI)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95th Percentile (SV95C) (Ambulatory Participants Only)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity Percent Predicted (FVC%p)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second % Predicted (FEV1%p)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR)(Baseline through Month 60)
- Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Inspiratory Pressure (MIP%p)(Baseline through Month 60)
- Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP)(Baseline through Month 60)
- Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Expiratory Pressure (MEP%p)(Baseline through Month 60)
- Part B: Number of Participants with TEAEs(Baseline through Month 60)
研究者
研究点 (1)
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