NCT06138639招募中1 期
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 30
- 主要终点
- Incidence of treatment-emergent adverse events (AEs)
研究概览
简要总结
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to < 7 years of age. Cohort 2 will include participants 7 to < 12 years of age. Cohort 3 will include participants 0 to < 4 years of age. Cohort 4 will include participants 12 to < 18 years of age. Cohort 5 will include participants 10 to < 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Cohort 1: 4 to <7 years of age
- •Cohort 2: 7 to <12 years of age
- •Cohort 3: 0 to < 4 years of age
- •Cohort 4: 12 to < 18 years of age
- •Cohort 5: 10 to < 18 years of age
- •Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in < 30 seconds:
- •Cohorts 1, 2, and 4: Ambulatory
- •Cohort 3: Either ambulatory or non-ambulatory
- •Cohort 5: Non-ambulatory, but having been previously ambulatory by history
- •Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
- •Negative for AAV antibodies.
- •Steroid regimen:
- •Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.
- •Cohort 3: N/A
- •Meet 10-meter walk/run time criteria
- •Meet time to rise from supine criteria
- •Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
- •Participant has body weight: ≤ 90 kg
排除标准
- •Treatment with dystrophin modifying drugs within 3 months prior to screening.
- •Current or prior treatment with an approved or investigational gene transfer drug.
- •Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
- •Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.
- •Other inclusion or exclusion criteria apply.
研究组 & 干预措施
Cohort 5: SGT-003
Experimental
All non-ambulatory participants from age 10 to < 18 years will receive a single IV infusion of SGT-003 on Day 1.
干预措施: SGT-003 (Genetic)
Cohort 3: SGT-003
Experimental
All participants from age 0 to < 4 years will receive a single IV infusion of SGT-003 on Day 1.
干预措施: SGT-003 (Genetic)
Cohort 2: SGT-003
Experimental
All ambulatory participants from age 7 to < 12 years will receive a single IV infusion of SGT-003 on Day 1.
干预措施: SGT-003 (Genetic)
Cohort 1: SGT-003
Experimental
All ambulatory participants from age 4 to < 7 years will receive a single IV infusion of SGT-003 on Day 1.
干预措施: SGT-003 (Genetic)
Cohort 4: SGT-003
Experimental
All ambulatory participants from age 12 to < 18 years will receive a single IV infusion of SGT-003 on Day 1.
干预措施: SGT-003 (Genetic)
结局指标
主要结局
Incidence of treatment-emergent adverse events (AEs)
时间窗: Day 360
Change from baseline in Microdystrophin Protein Levels
时间窗: Day 90
Microdystrophin expression evaluation in muscle biopsies
次要结局
- Change from baseline in North Star Ambulatory Assessment (NSAA) total score(Day 360, Day 540)
- Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters(Through Day 360 and Day 540)
- Change from baseline in 6-minute walk test (6MWT) distance(Day 360, Day 540)
- Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF)(Day 90, Day 360)
- Change from baseline in Microdystrophin Protein Levels(Day 360)
- Change from Baseline in Time to Rise Velocity(Day 360, Day 540)
- Change from baseline in Stride Velocity 95th Centile (SV95C)(Day 360, Day 540)
- Change from baseline in 10-meter walk/run velocity(Day 360, Day 540)
- Change from baseline in 4-stair climb velocity(Day 360, Day 540)
- Change from baseline in North Star Ambulatory Assessment (NSAA) total score(Day 360, Day 540)
- Change from baseline in 6-minute walk test (6MWT) distance(Day 360, Day 540)
- Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters(Through Day 360 and Day 540)
- Number of Participants with Clinically Significant Abnormalities in Vital Signs(Through Day 360 and Day 540)
- Number of Participants with Clinically Significant Abnormalities in Physical Examinations(Through Day 360 and Day 540)
- Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) or Echocardiography (ECHO)(Through Day 360 and Day 540)
研究者
研究点 (30)
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相关资讯
Solid Biosciences' SGT-003 Gene Therapy Shows Promise in Duchenne Muscular Dystrophy Trial- Solid Biosciences reported positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial of SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD).
- The trial showed an average microdystrophin expression of 110% in the first three patients, along with improvements in biomarkers indicating muscle health and resilience.
- SGT-003 was well-tolerated in the first six participants, with adverse events typical of AAV gene therapy and no serious safety concerns reported.
- Solid Biosciences plans to meet with the FDA to discuss a potential accelerated approval pathway for SGT-003 based on these encouraging early results.last yearSolid Biosciences Tracks Long-Term Cardiac Effects of SGT-001 Gene Therapy for Duchenne Muscular Dystrophy- Long-term follow-up data from the IGNITE-DMD trial indicate preserved cardiac function in most Duchenne muscular dystrophy (DMD) patients treated with SGT-001 gene therapy.
- The Phase 1/2 study assessed SGT-001, an AAV9 vector-based gene therapy delivering a microdystrophin gene to address the underlying cause of DMD.
- Solid Biosciences is shifting focus to SGT-003, a next-generation gene therapy for DMD, currently under evaluation in the INSPIRE-DUCHENNE clinical trial.
- SGT-003 leverages a novel capsid, AAV-SLB101, designed for more potent muscle transduction and contains a differentiated transgene.2 years ago