NCT05523167已完成2 期
A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 265
- 试验地点
- 365
- 主要终点
- Total improvement score (TIS); measured on a [0,100] scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).
研究概览
简要总结
This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study. Treatment response will be measured by Total improvement score (TIS).
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability to consent in the jurisdiction in which the study is taking place and capable of giving signed informed consent.
- •A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)
- •One of the following medical histories: Diagnosis of dermatomyositis (DM) or juvenile dermatomyositis (JDM), Diagnosis of polymyositis (PM) (including antisynthetase syndrome (ASyS)), Diagnosis of immune-mediated necrotizing myopathy (IMNM)
- •Diagnosed with active disease as defined by the presence of at least 1 of the following criteria: Abnormal levels of at least 1 of the following enzymes: creatine kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST), alanine aminotransferase (ALT), based on central laboratory results; Electromyography demonstrating active disease within the past 3 months; Active dermatomyositis (DM) skin rash; Muscle biopsy indicative of active idiopathic inflammatory myopathy (IIM) in the past 3 months; Magnetic resonance imaging within the past 3 months indicative of active inflammation
- •Muscle weakness
- •Receiving a permitted background treatment for idiopathic inflammatory myopathy.
- •Contraceptive use consistent with local regulations, where available, for individuals participating in clinical studies. Women of childbearing potential must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline before receiving investigational medicinal product (IMP).
排除标准
- •Any other known autoimmune disease that, in the investigator's opinion, would interfere with an accurate assessment of clinical symptoms of idiopathic inflammatory myopathy (IIM) or put the patient at undue risk
- •A history of malignancy unless considered cured by adequate treatment, with no evidence of recurrence for ≥ 3 years before the first administration of the investigational medicinal product (IMP). Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer ; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological finding of prostate cancer
- •Severe muscle damage
- •Glucocorticoid-induced myopathy that the investigator considers the primary cause of muscle weakness or permanent weakness linked to a non-idiopathic inflammatory myopathy (IIM) cause
- •Uncontrolled interstitial lung disease or any other uncontrolled idiopathic inflammatory myopathy (IIM) manifestation that, in the opinion of the investigator, would be likely to require treatment with prohibited medication during the study
- •Participant is pregnant or lactating or intends to become pregnant during the study.
研究组 & 干预措施
EFG PH20 SC
Experimental
participants receiving efgartigimod PH20 SC on top of background treatment
干预措施: EFG PH20 SC (Biological)
PBO PH20 SC
Placebo Comparator
participants receiving placebo PH20 SC on top of background treatment
干预措施: PBO (Other)
结局指标
主要结局
Total improvement score (TIS); measured on a [0,100] scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).
时间窗: phase 2: 24 weeks; phase 3: 52 weeks
Mean TIS
时间窗: phase 2: 24 weeks; phase 3: 52 weeks
The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).
次要结局
- Percentage of participants with TIS ≥ 20(phase 2: 24 weeks; phase 3: 52 weeks)
- Percentage of participants with TIS ≥ 40(phase 2: 24 weeks; phase 3: 52 weeks)
- Change in manual muscle testing-8 (MMT8) score(phase 2: 24 weeks; phase 3: 52 weeks)
- Change in Patient Global Assessment of Disease Activity (PGA)(phase 2: 24 weeks; phase 3: 52 weeks)
- Change in Physician Global Assessment of Disease Activity (MDGA)(phase 2: 24 weeks; phase 3: 52 weeks)
- Proportion of participants achieving target dose of ≤ 5 mg (prednisone equivalent)(Phase 3: 52 weeks)
- Time to reach TIS ≥ 20 (first "minimal clinical improvement")(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Time to reach TIS ≥ 40 (first "moderate clinical improvement")(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Percentage of participants with TIS ≥ 20(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Percentage of participants with TIS ≥ 40(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Change in MMT8 score(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Change in Patient Global Assessment of Disease Activity (PGA)(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Change in Physician Global Assessment of Disease Activity (MDGA)(phase 2: up to 24 weeks; phase 3: up to 52 weeks)
- Proportion of participants who have at least moderate improvement (≥40) in TIS at week 52 and adhere to an oral prednisone dosage of ≤5 mg/day (or equivalent) from week 44 onward(Phase 3: up to 52 weeks)
- Change in CK abnormality grades at week 52(Phase 3: up to 52 weeks)
- Change in CDASI activity score at week 52(Phase 3: up to 52 weeks)
研究者
研究点 (365)
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