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)

Status: Recruiting
Location: See all (5) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 16
Healthy Volunteers: f
View:

• 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

Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
Maryland
Johns Hopkins University
NOT_YET_RECRUITING
Baltimore
Pennsylvania
University of Pittsburgh
NOT_YET_RECRUITING
Pittsburgh
Texas
University of Texas Health Science Center at Houston
ACTIVE_NOT_RECRUITING
Houston
Washington
University of Washington
ACTIVE_NOT_RECRUITING
Seattle
Contact Information
Primary
James Andrews, MD
jaandrews@uabmc.edu
(205) 934-1564
Backup
Hannah E Howell, MS
heburns@uabmc.edu
205-996-6552
Time Frame
Start Date: 2025-10-27
Estimated Completion Date: 2027-06-30
Participants
Target number of participants: 12
Treatments
Experimental: Intravenously Administered Pooled Human Immunoglobulin (IVIG)
Participants will receive intravenously administered pooled human immunoglobulin (IVIG) 2g/kg every 4 weeks for 24 weeks.
No_intervention: 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.
Related Therapeutic Areas
Sponsors
Leads: University of Alabama at Birmingham
Collaborators: Octapharma USA, Inc., National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

This content was sourced from clinicaltrials.gov