The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Mediated Necrotizing Myopathy

Phase 2

Not yet recruiting

• Conditions: Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy
• Interventions: Biological: Intravenously administered pooled human immunoglobulin (IVIG)

• Registration Number: NCT06599697
• Lead Sponsor: University of Alabama at Birmingham

Brief Summary

This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of IVIG in anti-HMGCR IMNM. Planned enrollment is 12 individuals with active anti-HMGCR IMNM meeting inclusion and exclusion criteria. Assuming 20% drop-out, we anticipate 10 participants will complete all study assessments. Enrolled participants will be randomized 1:1 to either IVIG 2g/kg or placebo (0.9% NaCl 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 ofthe trial, all participants, except those who were randomized to IVIG and met the clinical deterioration criteria, will be offered to continue on to an open-label extension phase in which they will receive IVIG at weeks 12, 16, and 20. They will then return at week 24 for a final non-infusion visit to reassess safety, tolerability, and efficacy outcome

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-12
• Study First Submitted QC: 2024-09-12
• Study First Posted: 2024-09-19
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-03
• Last Update Posted: 2025-05-07
• Study Start: 2025-07-01
• Primary Completion: 2026-12-30
• Study Completion: 2027-06-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• 16 years of age or older
• Anti-HMGCR antibody positive
• Serum creatine kinase > 5x upper limit of normal
• Anti-HMGCR Immune Mediated Necrotizing Myopathy disease duration < 36 months at screening
• No moderate or severe respiratory or swallowing dysfunction due to Immune Mediated Necrotizing Myopathy at screening
• No history of dermatomyositis rash

Exclusion Criteria

• Oral glucocorticoid daily dose > 15mg at screening
• Change in oral glucocorticoid dose < 2 weeks prior to screening
• Prior IVIG treatment for anti-HMGCR Immune Mediated Necrotizing Myopathy
• > 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 treatment within 6 months prior to screening
• Plasma exchange, cyclophosphamide, or biologic immunosuppressive medication within 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 within 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 Pulmonary Embolism score of 4 or more at screening
• Wells' Criteria for Deep Vein Thrombosis score of 2 or more at 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Intravenously Administered Pooled Human Immunoglobulin (IVIG)	Intravenously administered pooled human immunoglobulin (IVIG)	Participants will receive intravenously administered pooled human immunoglobulin (IVIG) 2g/kg every 4 weeks for 24 weeks.

Primary Outcome Measures

Name	Time	Method
Percentage change in serum creatine kinase (CK)	Week 0 to 12	Primary Efficacy Outcome

Trial Locations

Locations (1)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States