The MIGHT Trial - An Exploratory Clinical Trial of Intravenous Immunoglobulin (IVIG) in Anti-3-Hydroxy-3-Methylglutaryl-CoA Reductase (HMGCR) Immune Mediated Necrotizing Myopathy (IMNM)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 5
- 主要终点
- Percentage change in serum creatine kinase (CK)
研究概览
简要总结
This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated necrotizing myopathy (IMNM). Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Assuming 20% drop-out, the investigators anticipate 10 participants will complete all study assessments. Enrolled participants will be randomized 1:1 to either IVIG 2g/kg or placebo (0.9% sodium chloride at equivalent volume) at weeks 0, 4, and 8. The primary efficacy and co-primary safety and tolerability endpoints will be assessed at week 12. After the randomized phase of the trial, all participants will be offered to continue on to an open-label extension phase in which participants will receive IVIG at weeks 12, 16, and 20. Participants will then return at week 24 for a final non-infusion visit to reassess safety, tolerability, and efficacy outcome.
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)
盲法说明
Using an online randomization tool (e.g. REDCap), 12 total participants will be randomized 1:1 in blocks of 4 to receive either intravenously administered pooled human immunoglobulin (IVIG) 2g/kg every 4 weeks or placebo (0.9% sodium chloride solution at equivalent volume to corresponding IVIG weight-based dose). The home infusion research pharmacy (CSI Pharmacy) will assign treatment status using the randomization tool and will prepare IVIG or placebo for home infusion nursing staff in a blinded fashion.
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age > 16 years
- •Anti-HMGCR antibody positive
- •MMT-8 score < 142 (range 0-160)
- •Serum CK > 5x upper limit of normal
- •Anti-HMGCR IMNM disease duration < 36 months at screening
- •No moderate or severe respiratory or swallowing dysfunction due to anti-HMGCR IMNM at screening
- •No history of dermatomyositis rash
- •Must reside in a state with a participating research site
排除标准
- •Oral glucocorticoid (GC) daily dose > 15mg at screening
- •Change in oral GC dose < 2 weeks prior to screening
- •Prior IVIG treatment for anti-HMGCR IMNM
- •>1 oral conventional synthetic DMARD (e.g. methotrexate, mycophenolate mofetil, azathioprine) use at screening
- •Change in concomitant DMARD dose < 4 weeks prior to screening
- •Rituximab < 6 months prior to screening
- •Plasma exchange, cyclophosphamide, or biologic immunosuppressive medication < 3 months prior to screening
- •Use of statin medication at screening
- •History of anaphylactic reaction to IVIG
- •History of angina pectoris, myocardial infarction, transient ischemic attack, or stroke < 12 months prior to screening
- •Females of child-bearing potential who are pregnant, breastfeeding, or are unwilling to practice a highly effective method of contraception during the study
- •Wells Criteria for DVT score of 2 or more at screening
- •Wells Criteria for PE score of 4 or more at screening
- •Weight >120kg
- •History of cancer (excluding non-melanomatous skin cancer) < 5 years prior to screening
- •History of pulmonary embolism or deep venous thromboembolism < 3 years prior to screening
- •History of hyperviscosity or hypercoagulable state
- •Currently receiving anti-coagulation therapy (vitamin K antagonists, non-vitamin K oral anticoagulants [e.g. dabigatran, rivaroxaban, apixaban], parenteral anticoagulants [e.g. fondaparinux]. Note that oral anti-platelet agents are allowed (e.g. aspirin, clopidogrel, ticlopidine).
- •Glomerular filtration rate (GFR) <60mL/min at the time of screening
- •Any medical condition which, in the investigator's judgment, makes participation in the clinical trial unadvisable or which would interfere with evaluation of the study treatment.
研究组 & 干预措施
Intravenously Administered Pooled Human Immunoglobulin (IVIG)
Participants will receive intravenously administered pooled human immunoglobulin (IVIG) 2g/kg every 4 weeks for 24 weeks.
干预措施: Intravenously administered pooled human immunoglobulin (IVIG) (Biological)
Placebo
Participants will receive an infusion of 0.9% sodium chloride solution every 4 weeks for 16 weeks at equivalent volume to corresponding IVIG weight-based dose.
结局指标
主要结局
Percentage change in serum creatine kinase (CK)
时间窗: Week 0 to 12
Primary Efficacy Outcome
次要结局
未报告次要终点
研究者
James Andrews
Assistant Professor of Medicine
University of Alabama at Birmingham