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临床试验/NCT06747273
NCT06747273终止1 期

A Phase 1b, Multicenter, Single Dose Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Subjects in the United States

Sarepta Therapeutics, Inc.2 个研究点 分布在 1 个国家目标入组 4 人开始时间: 2025年1月9日最近更新:
适应症
干预措施
相关药物

试验速览

阶段
1 期
状态
终止
入组人数
4
试验地点
2
主要终点
Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)

研究概览

简要总结

The primary objective of this study is to evaluate the safety of SRP-9004.

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

入选标准

  • Ambulatory participants, defined as able to walk without assistive aid, 10MWR <30 seconds, and NSAD total score ≥25; non-ambulatory participant, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥
  • Ambulatory participants must be 4 to 20 years of age and the non-ambulatory participant must be ≥4 years of age.
  • All participants must be ≤70 kilograms
  • Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic α-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to screening.
  • Able to cooperate with muscle testing.
  • Participants must have adeno-associated virus (AAV) serotype Rh74 (rh74) antibody titers <1:400 (that is, not elevated) as determined by an enzyme-linked immunosorbent assay (ELISA).

排除标准

  • Left ventricular ejection fraction <40% or clinical signs and/or symptoms of cardiomyopathy
  • FVC ≤40% of predicted value and/or requirement for nocturnal ventilation
  • Any other clinically significant illness, including neuromuscular (other than limb girdle muscular dystrophy type 2D/R3 [LGMD2D/R3]), that in the opinion of the Investigator might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
  • Other inclusion/exclusion criteria apply.

研究组 & 干预措施

SRP-9004

Experimental

Participants will receive a single dose of SRP-9004 on Day 1.

干预措施: SRP-9004 (Drug)

结局指标

主要结局

Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)

时间窗: Up to 60 months

次要结局

  • Change from Baseline Through Day 60 in Quantity of Alpha-sarcoglycan (α-SG) Protein Expression(Baseline, Day 60)
  • Change from Baseline Through Month 24 in Quantity of α-SG Protein Expression(Baseline, Month 24)
  • Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Time to Rise From Floor(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Time to Ascend 4 Steps(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Time to Complete 10-meter Walk/Run (10MWR)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR)(Baseline, Month 60)
  • Change from Baseline through Month 60 in Creatine Kinase (CK) Level(Baseline, Month 60)
  • Time to Change of Disease Milestones (Loss of Ambulation)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imagining (MRI) Findings(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95% (SV95C) (Ambulatory Participants Only)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FVC Percentage (%) Predicted(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FEV1 % Predicted(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP)(Baseline, Month 60)
  • Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP)(Baseline, Month 60)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (2)

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