A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 (Subcutaneous Immunoglobulin, Hizentra®) in Adults With Dermatomyositis (DM) - The RECLAIIM Study
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- CSL Behring
- 入组人数
- 134
- 试验地点
- 76
- 主要终点
- Responder Rate
研究概览
简要总结
This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of IgPro20 (subcutaneous Ig) treatment in adult subjects with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of IgPro20 subcutaneous (SC) doses in comparison to placebo in adult subjects with DM, as measured by responder status based on Total Improvement Score (TIS) assessments.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects ≥ 18 years of age
- •Diagnosis of at least probable idiopathic inflammatory myopathies (IIM) per European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) Classification Criteria which includes confirmation of dermatomyositis (DM) rash/manifestation, disease activity defined by presence of DM rash / manifestation or an objective disease activity measure
- •Disease severity defined by Physician global activity visual analog scale (VAS) with a minimum value of 2.0 cm on a 10 cm scale and MMT-8 ≤ 142 or CDASI total activity score ≥
- •Corticosteroid daily dose less than that or equal to 20 mg prednisolone equivalent
排除标准
- •Cancer-associated myositis
- •Evidence of active malignant disease or malignancies diagnosed within the previous 5 years
- •Physician Global Damage score ≥ 3, or clinically relevant improvement between Screening Visit and Baseline
研究组 & 干预措施
IgPro20
human immunoglobulin G administered subcutaneously
干预措施: human immunoglobulin G (Drug)
Placebo
human albumin solution administered subcutaneously
干预措施: Placebo (Drug)
结局指标
主要结局
Responder Rate
时间窗: At Week 25
A responder was defined as a participant with a TIS \>= 20 points at Week 25 and at least 1 of the previous scheduled visits (Week 17 or 21), who completes 24 weeks of randomized IMP treatment without the use of rescue corticosteroid treatment. The TIS was a sum response criterion which incorporates 6 weighted international myositis assessment and clinical studies (IMACS) core set measures (CSMs) including Physician and Patient Global Disease Activity (PGA), Manual Muscle Testing-8 (MMT-8), Health Assessment Questionnaire, Muscle Enzyme, and Extramuscular Global Activity (EGA). Thresholds for minimal, moderate, and major improvement were \>= 20, \>= 40, and \>= 60 points, respectively on the TIS. Percentage of responders at Week 25 based on TIS are reported here. Multiple imputation (MI) was used to impute missing values for participants who discontinued due to the military activities in Ukraine.
次要结局
- Mean TIS(At Week 25)
- Mean Changes From Baseline in MMT-8(From Baseline to Week 25)
- Mean Changes From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index Total Activity Score (CDASI-A)(From Baseline to Week 25)
- Percentage of Participants Who Are Able to Reduce the Oral Corticosteroid Dose by >= 25%(At Week 25)
- Period 1: Mean TIS at Each Visit(At Week 5, 9, 13, 17, 21, and 25)
- Period 2: Mean TIS at Each Visit(Week 25, 29, 33, 37, 41, 45, 49, and 53)
- Period 1: Percentage of Participants Achieving TIS ≥ 20, ≥ 40 and ≥ 60 Points(At Week 5, 9, 13, 17, 21, and 25)
- Period 2: Percentage of Participants Achieving TIS ≥ 20, ≥ 40 and ≥ 60 Points(Week 25, 29, 33, 37, 41, 45, 49, and 53)
- Period 1: Time to First Achieving TIS >= 20, >= 40, and >= 60 Points on the TIS(Up to Week 25)
- Period 1 and 2: Time to First Achieving TIS >= 20, >= 40, and >= 60 Points Improvement on the TIS(Up to Week 53)
- Mean Changes From Baseline in Individual CSMs (Except Muscle Enzymes) and CDASI(From Baseline to Week 25)
- Mean Changes From Week 25 in Individual CSMs (Except Muscle Enzymes) and CDASI(From Week 25 to Week 53)
- Period 1: Number of Participants Meeting Definition of Worsening (DOW) at Least Once, Twice, or > Twice(Up to Week 25)
- Period 1: Percentage of Participants Meeting DOW at Least Once, Twice, or > Twice(Up to Week 25)
- Period 2: Number of Participants Meeting DOW at Least Once, Twice, or > Twice(From Week 25 up to Week 53)
- Period 2: Percentage of Participants Meeting DOW at Least Once, Twice, or > Twice(From Week 25 up to Week 53)
- Time to Meeting DOW for the First Time(Up to Week 53)
- Number of Participants Meeting DOW and Receiving Rescue Steroid Treatment(Up to Week 25)
- Percentage of Participants Meeting DOW and Receiving Rescue Steroid Treatment(Up to Week 25)
- Number of Participants Who Start Oral Corticosteroid Dose Taper(Up to Week 25)
- Percentage of Participants Who Start Oral Corticosteroid Dose Taper(Up to Week 25)
- Number of Participants Who Are Able to Reduce the Oral Corticosteroid Dose by >= 25%, >= 50%, >= 75%(Up to Week 25 and 52)
- Percentage of Participants Who Are Able to Reduce the Oral Corticosteroid Dose by >= 25%, >= 50%, >= 75%(Up to Week 25 and 52)
- Percentage of Participants Receiving Rescue Corticosteroid Treatment(Up to Week 25)
- Percentage of Participants Whose Rescue Corticosteroid Treatment is Tapered(Up to Week 25)
- Time to First Intake of Rescue Corticosteroid Treatment(Up to Week 25)
- Number of Participants Having at Least 1 Level, 2 Levels, and More Than 2 Levels of Improvement From Baseline in Mobility, Self-care, and Usual Activities Domains of EuroQoL 5-Dimension Questionnaire (EQ-5D-5L)(From Baseline to Week 13 and 25)
- Percentage of Participants Having at Least 1 Level, 2 Levels, and More Than 2 Levels of Improvement From Baseline in Mobility, Self-care, and Usual Activities Domains of EQ-5D-5L(From Baseline to Week 13 and 25)
- Number of Participants Having no Reduction in Levels, at Least 1 Level, 2 Levels, and More Than 2 Levels of Improvement From Week 25 in Mobility, Self-care, and Usual Activities Domains of EQ-5D-5L(From Week 25 to 41 and 53)
- Percentage of Participants Having no Reduction in Levels, at Least 1 Level, 2 Levels, and More Than 2 Levels of Improvement From Week 25 in Mobility, Self-care, and Usual Activities Domains of EQ-5D-5L(From Week 25 to 41 and 53)
- Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Related TEAEs and Serious TEAEs(Up to 5 years)
- Annualized Rate of TEAEs, Related TEAEs and Serious TEAEs Per Time at Risk(Up to 5 years)
- Annualized Rate of Mild, Moderate, and Severe TEAEs Per Time at Risk(Up to 5 years)