NCT07160634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
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 80
- 试验地点
- 7
- 主要终点
- Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540
研究概览
简要总结
This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 7 Years 至 11 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participant is ambulatory.
- •Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
- •Negative for antibodies against adeno-associated virus serotype 9 (AAV9).
- •On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (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.
排除标准
- •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.
- •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.
- •Other Inclusion/Exclusion criteria to be applied as per protocol.
研究组 & 干预措施
SGT-003 in Part 1, Placebo in Part 2
Experimental
Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria.
干预措施: Placebo (Drug)
Placebo in Part 1, SGT-003 in Part 2
Experimental
Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2 per protocol-defined eligibility criteria.
干预措施: Placebo (Drug)
SGT-003 in Part 1, Placebo in Part 2
Experimental
Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and may receive a single IV infusion of matching Placebo in Part 2 per protocol-defined eligibility criteria.
干预措施: SGT-003 (Drug)
Placebo in Part 1, SGT-003 in Part 2
Experimental
Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2 per protocol-defined eligibility criteria.
干预措施: SGT-003 (Drug)
结局指标
主要结局
Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540
时间窗: Baseline, Day 540
次要结局
- Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540(Baseline, Day 540)
- Change From Baseline in 4-Stair Climb (4SC) Velocity (tasks/s) at Day 540(Baseline, Day 540)
- Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540(Baseline, Day 540)
- Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540(Baseline, Day 540)
- Cumulative Loss of Function in NSAA Items at Day 540(At Day 540)
- Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90(Baseline, Day 90)
- Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90(Baseline, Day 90)
- Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540(Baseline, Day 540)
- Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540(Baseline, Day 540)
- Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540(Baseline, Day 540)
- Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540(Baseline, Day 540)
- Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540(Baseline, Day 540)
- Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540(Baseline, Day 540)
- Cumulative Loss of Function in NSAA Items at Day 540(At Day 540)
- Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90(Baseline, Day 90)
- Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90(Baseline, Day 90)
- Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540(Baseline, Day 540)
- Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540(Baseline, Day 540)
- Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540(Baseline, Day 540)
- Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540(Baseline, Day 540)
- Change from Baseline in Global Longitudinal Strain (GLS) at Day 540(Baseline, Day 540)
- Change from Baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540(Baseline, Day 540)
- Change From Baseline in 4-Stair Climb (4SC) Velocity (Tasks/s) at Day 540(Baseline, Day 540)
- Change from Baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540(Baseline, Day 540)
- Change from Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540(Baseline, Day 540)
- Change from Baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540(Baseline, Day 540)
- Change from Baseline in Left Ventricular Ejection Fraction (LVEF) at Day 540[(Baseline, Day 540)
- Change From Baseline in Viral Shedding as Measured by Polymerase Chain Reaction (PCR) at Day 360(Baseline, Day 360)
- Change From Baseline in Anti-Adeno-Associated Virus (AAV) Antibody Titers at Day 540(Baseline, Day 540)
- Change from Baseline in Microdystrophin T-cell Reactivity by Enzyme-Linked Immunospot at Day 540(Baseline, Day 540)
- Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Adverse Events of special interest (AESIs), and clinically significant changes in Electrocardiogram (ECG) and Echocardiography (ECHO)(From first dose up to Day 540)
研究者
研究点 (7)
Loading locations...
相似试验
终止
3 期
The Safety and Efficacy of Brexpiprazole as Adjunctive Therapy in the Treatment of Major Depressive DisorderMajor Depressive DisorderNCT03487198Otsuka Beijing Research Institute65
已完成
3 期
A Study of Atezolizumab and Paclitaxel Versus Placebo and Paclitaxel in Participants With Previously Untreated Locally Advanced or Metastatic Triple Negative Breast Cancer (TNBC)Triple-Negative Breast CancerNCT03125902Hoffmann-La Roche653
进行中（未招募）
3 期
Study of Emactuzumab for Tenosynovial Giant Cell Tumor (TGCT)NCT05417789SynOx Therapeutics Limited128
进行中（未招募）
3 期
Study to Assess Efficacy and Safety of M72/AS01E-4 Mycobacterium Tuberculosis (Mtb) Vaccine in Adolescents and AdultsTuberculosisNCT06062238Gates Medical Research Institute20,080
已完成
3 期
Certolizumab Pegol in Subjects With Active Axial SpondyloarthritisSpondyloarthropathiesNCT01087762UCB BIOSCIENCES GmbH325