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

Conditions

• Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy

Eligibility Criteria

Sex

ALL

Age

16 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Intravenously administered pooled human immunoglobulin (IVIG) — BIOLOGICAL
  IVIG 2g/kg every 4 weeks for 12 weeks (3 doses)

Study Details

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.

Key Dates

Start date

Oct 27, 2025

Status verified

Feb 2026

Primary completion

Dec 30, 2026

Completion

Jun 30, 2027

Study Design

Enrollment

12 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• 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.

Primary Outcome Measure

Percentage change in serum creatine kinase (CK) [ Time Frame: Week 0 to 12 ]

Central Contacts

• James Andrews, MD
  (205) 934-1564
  [email protected]
• Hannah E Howell, MS
  205-996-6552
  [email protected]

Locations (5)

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