2025-522949-22-00招募中3 期
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 62
- 试验地点
- 12
- 主要终点
- Change from baseline in time to rise velocity at Day 540
研究概览
简要总结
To investigate the efficacy of a single intravenous dose of SGT-003 compared to placebo by assessing change in muscle function.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participant 7 to <12 years of age.
- •Participant is able to understand and comply with all study procedures as appropriate by age and has a parent(s) or legal guardian(s) (i.e., legally authorized representative [LAR]) who is (are) able to understand and comply with the study procedure requirements. Be willing to provide informed assent and have an LAR(s) who is (are) willing to provide written informed consent for the participant to participate in the study.
- •If participant is of reproductive potential, participant and partner of childbearing potential are willing to use 2 highly effective forms of contraception for 12 months following study drug administration.
- •Participant is ambulatory. Ambulatory is defined as “being able to walk without the use of an assistive device.”
- •Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype.
- •Negative for AAV antibodies.
- •On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 mg/kg/day deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice.
- •Meet 10-meter walk/run time criteria.
- •Meet time to rise from supine criteria.
- •Participant has bodyweight ≤50 kg
- •Participant is genetically male.
排除标准
- •Prior or ongoing medical condition, medical history, or physical finding that in the Investigator's opinion could adversely affect the safety of the participant, make it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
- •History of severe hypersensitivity reactions, including anaphylaxis, to the product or its components.
- •Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.
- •Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment.
- •Any active infection.
- •Major surgery within 3 months prior to recruitment or planned surgery any time during this study that would have the potential to interfere with the ability or performance on outcome measures.
- •Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive, of the DMD gene as documented by a genetic report.
- •Sponsor employees and their family members are ineligible to participate in this study.
- •Known liver disease, evidence of active viral hepatitis, or abnormal liver function.
- •Abnormal renal function
- •Clinically significant abnormalities of coagulation
- •Impaired cardiovascular function
- •Pulmonary function predictive of or requiring the use of daytime ventilatory support outside of acute illnesses.
研究组 & 干预措施
Normal saline (0.9% sodium chloride) for infusion
Placebo
干预措施: Normal saline (0.9% sodium chloride) for infusion (Drug)
结局指标
主要结局
Change from baseline in time to rise velocity at Day 540
Change from baseline in time to rise velocity at Day 540
次要结局
- Change from baseline in % predicted forced vital capacity (FVC) at Day 540
- Incidence of serious adverse events through Day 540
- Incidence of adverse events of special interest through Day 540
- Incidence of clinically significant changes in ECGs through Day 540
- Incidence of clinically significant changes in ECHOs through Day 540
- Change from baseline in the PODCI Global score at Day 540.
- Change from baseline in % predicted peak expiratory flow (PEF) at Day 540
- Change from baseline in % predicted forced expiratory volume in 1 second (FEV1) at Day 540
- Incidence of treatment-emergent adverse events through Day 540
- Change from baseline in microdystrophin tissue distribution at Day 90
- Change from baseline in stride velocity 95th centile (SV95C) by activity monitoring using the Syde wearable device at Day 540
- Change from baseline in 10-meter walk/run velocity at Day 540
- Change from baseline in 4-stair climb velocity at Day 540
- Change from baseline in absolute NSAA score at Day 540
- Cumulative loss of function in North Star Ambulatory Assessment (NSAA) items at Day 540
- Change from baseline in microdystrophin protein levels at Day 90
研究者
Amber Conklin
Scientific
Solid Biosciences Inc.
研究点 (12)
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