Recruiting
Phase 2

IVIG

Sponsor:

University of Alabama at Birmingham

Code:

NCT06599697

Conditions

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

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Interventions

Intravenously administered pooled human immunoglobulin (IVIG)

Study Details

Brief 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.

Conditions

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

Study ID

NCT06599697

Start date

Oct 27, 2025

Status verified date

Aug, 2026

Completion date

Jun 30, 2027

Anticipated

Primary completion date

Dec 30, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 16+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.

Study Design

Enrollment

12 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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.

Interventions

Intravenously administered pooled human immunoglobulin (IVIG)

IVIG 2g/kg every 4 weeks for 12 weeks (3 doses)

Primary outcome measure

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

Central Contacts and Locations

Central contacts

James Andrews, MD

(205) 934-1564jaandrews@uabmc.edu

Hannah E Howell, MS

205-996-6552heburns@uabmc.edu

Locations

University of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

James Andrews, MD

(205) 934-1564jaandrews@uabmc.edu

Hannah E Howell, MS

205-996-6552heburns@uabmc.edu

Principal Investigator:

James Andrews, MD

University of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15260

Contacts

Rohit Aggarwal, MD, MS

412-624-4141aggarwalr@upmc.edu

Diane C Koontz

dik4@pitt.edu

University of Texas Health Science Center at Houston

Recruiting

Houston, Texas, United States, 77030

Contacts

Eleni Tiniakou, MD

713-500-4435eleni.tiniakou@uth.tmc.edu

Enrique Rodriguez

Enrique.Rodriguez@uth.tmc.edu

More Information

Sponsor

University of Alabama at Birmingham

Last update posted

Aug 5, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by University of Alabama at Birmingham on 2026-08-05.