NCT06450639进行中（未招募）2 期
A Phase II Multicenter, Open-label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Pediatric Patients With Duchenne Muscular Dystrophy (SHIELD DMD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 21
- 主要终点
- Group 2: Change From Baseline to Week 52 in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score Measured by Dual-energy X-ray Absorptiometry (DEXA)
研究概览
简要总结
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory participants with DMD aged ≥ 8 to < 18 years old receiving corticosteroid therapy.
详细描述
Participants will be included in two groups: ambulatory participants with fractures and non-ambulatory participants with or without a history of fractures (Group 1) and ambulatory participants who are fracture-naïve (Group 2) at baseline. The study will assess the potential of satralizumab to improve bone fragility and to increase muscle function. A weight-tier-based dose of satralizumab will be given by subcutaneous (SC) injection every 4 weeks (Q4W).
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
- 8 Years 至 17 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Signed Informed Consent Form (ICF) and signed Assent Form when appropriate
- •Male at birth
- •A definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test
- •Age ≥ 8 and < 18 years at the time of signing ICF
- •Group 1 participants are required to meet the following criteria: - Ambulatory (defined as able to walk independently without assistive devices) with a prior history of fractures: a) Prior history of low-trauma fracture defined as: evidence of at least one prevalent vertebral compression fracture of Genant Grade 1 or 2 (or radiographic signs of vertebral fractures [VF]) or history of at least one low-trauma long-bone fracture (upper or lower extremity) or b) Non-ambulatory, characterized as being non-ambulatory for a minimum of 6 months with onset of non-ambulatory status defined as participant- or caregiver-reported age of continuous wheelchair use, approximated to the nearest month, and an North Star Ambulatory Assessment (NSAA) walk score of "0" and inability to perform the 10-Meter Walk/Run (10 MWR) at the baseline visit, with or without fractures
- •Group 2 participants are required to meet the following criteria: - Be fracture-naïve, defined as: no history of prior low-trauma fractures before the baseline visit nor any radiological findings indicative of prevalent VF at the screening visit - Be ambulatory defined as able to walk independently without assistive devices - Age ≥ 8 to < 12 years old at the time of screening
- •Daily oral corticosteroids
排除标准
- •Major surgery (e.g., spinal surgery) within 3 months prior to baseline or planned surgery or procedure that would interfere with the conduct of the study for any time during this study
- •Presence of any clinically significant illness
- •Has serological evidence of current, chronic, or active human immunodeficiency virus (HIV), tuberculosis (TB), hepatitis C virus (HCV), or hepatitis B virus (HBV) infection
- •Has a symptomatic infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to baseline
- •Body weight at screening < 20 or > 100 kilograms (kg)
- •Evidence of a severe VF (defined as Grade 3), assessed by radiographic imaging at screening and quantified using the Genant semiquantitative method
- •Treatment with prohibited therapies as defined by the protocol
- •Has received a live or live attenuated virus vaccine within 6 weeks of the baseline visit or expects to receive a live or live attenuated virus vaccine during the study
- •Has abnormal laboratory values considered clinically significant as defined by the protocol
- •Any medical condition that might interfere with the evaluation of LS BMD, such as severe scoliosis or spinal fusion
- •Participant has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the investigator
- •Participant has an allergy or hypersensitivity to the study medication or to any of its constituents. Other protocol defined inclusion and exclusion criteria may apply
研究组 & 干预措施
Satralizumab
Experimental
Satralizumab will be administered SC in the abdominal or femoral region on Day 1, Weeks 2 and 4 (loading doses) and then Q4W from Weeks 8 until the study completion (maintenance doses).
干预措施: Satralizumab (Drug)
结局指标
主要结局
Group 2: Change From Baseline to Week 52 in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score Measured by Dual-energy X-ray Absorptiometry (DEXA)
时间窗: Baseline to Week 52
BMD of the LS is measured using DEXA
Group 2: Change From Baseline to Week 24 in Lumbar Spine (LS) Bone Mineral Density (BMD) Z-score Measured by Dual-energy X-ray Absorptiometry (DEXA)
时间窗: Baseline up to Week 24
BMD of the LS is measured using DEXA.
次要结局
- All Participants: Change From Baseline to Weeks 24, 52, and 104 in LS BMD Z-score Measured by DEXA(Baseline to Week 24, Week 52 and Week 104)
- Group 2: Change From Baseline to Weeks 24 and 104 in LS BMD Z-score Measured by DEXA(Baseline to Week 24 and Week 104)
- Group 2: Change From Baseline to Weeks 24, 52 and 104 in Total Body Less Head Bone Mineral Density (TBLH BMD) Z-score Measured by DEXA(Baseline to Week 24, Week 52 and Week 104)
- Group 2: Change From Baseline to Weeks 24, 52 and 104 in Total Hip BMD Z-score Measured by DEXA(Baseline to Week 24, Week 52 and Week 104)
- Group 2: Change From Baseline to Weeks 12, 24 and 52 in Circulating Bone Metabolism Biomarkers(Baseline to Week 12, Week 24 and Week 52)
- All Participants: Change From Baseline to Weeks 12, 24 and 52 in Circulating Bone Metabolism Biomarkers(Baseline to Week 12, Week 24, and Week 52)
- All Participants: Mean Number Per Participants of New Low-trauma Long-bone or Vertebral Fractures (VF)(Baseline to Week 52 and Week 104)
- All Participants: Percentage of Participants With New Low-trauma Long-bone or VF(Baseline to Week 52 and Week 104)
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)(Up to approximately 30 Months)
- Percentage of Participants With Serious Adverse Events (SAEs)(Up to approximately 30 Months)
- Percentage of Participants With Adverse Events of Special Interest (AESIs)(Up to approximately 30 Months)
- Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Week 104(Up to Week 104)
- Apparent Clearance of Satralizumab(Up to Week 104)
- Apparent Volume of Distribution of Satralizumab(Up to Week 104)
- Area Under the Concentration-time Curve of Satralizumab(Up to Week 104)
- Percentage of Participants With Anti-drug Antibodies (ADAs) at Baseline and During the Study(Up to approximately 30 Months)
- Percentage of Participants With Adverse Events of Special Interest (AESIs)(Up to 90 weeks)
- Observed Serum Concentration of Satralizumab at Specified Trough Timepoints up to Study End(Up to 90 weeks)
- Apparent Clearance (CL) of Satralizumab(Up to 90 weeks)
- All Participants: Change From Baseline to Week 24 in LS BMD Z-score Measured by DEXA(Baseline up to Week 24)
- Group 2: Change From Baseline to Week 24 in Total Body Less Head (TBLH) BMD Z-score Measured by DEXA(Baseline up to Week 24)
- Group 2: Change From Baseline to Week 24 in Total Hip BMD Z-score Measured by DEXA(Baseline up to Week 24)
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)(Up to 90 weeks)
- Percentage of Participants With Serious Adverse Events (SAEs)(Up to 90 weeks)
- Apparent Volume of Distribution (Vd) of Satralizumab(Up to 90 weeks)
- Area Under the Concentration-time Curve (AUC) of Satralizumab(Up to 90 weeks)
- Percentage of Participants With Anti-drug Antibodies (ADAs) to Satralizumab at Baseline and During the Study(Up to 90 weeks)
研究者
研究点 (21)
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