Purpose:  This randomized double blind study hopes to determine whether the immunoglobulin (IVIG) can be used as a standalone drug to treat HMGCR Myopathy.  N=10

Patient Population:  For persons recently diagnosed with AntiHMG Auto-Antibody Positive Necrotizing Myopathy (HMGCR Myopathy). 

Locations:  2 Sites:  1) University of Washington Medical Center at the UW campus in Seattle  WA       2)   Johns Hopkins, Baltimore MD

UW researcher:  Principal Investigator is James Andrews, MD, a practicing Rheumatologist at the University of Washington

Study Overview: The study lasts for 12 weeks.  During that time participants will not take any other medications such as steroids to manage their HMGCR symptoms.

There are 4 main visits.  Two of those visits are infusion visits and may take up to 4 days (approximately 4 -6 hours each day) to complete the infusion.  This is dependent on participant’s weight and tolerance of the IVIG infusion.  Participant vitals will be monitored throughout the infusion and various lab tests will be done throughout the study to monitor the participant’s tolerance of the IVIG treatment.

Study Procedures include:  Physical Exams, 4 blood draws, Urine pregnancy test (if applicable), IVIG infusions, Muscle Strength Testing, and Questionnaires

Inclusion Criteria: 

  • A positive Anti-HMGCR lab result within 12 months prior to the participant signing the Informed Consent and entering Screening. 
  • Age ≥ 18 years
  • Demonstrable proximal muscle weakness: score of <135 on the Proximal Manual Muscle Strength Testing 8-Muscle Group Assessment (MMT-8) (range 0-160) (2, 3).
  • Serum creatinine kinase (CK) more than 5 times the upper limit of normal
  • Muscle biopsy will not be required for eligibility in order to minimize the time to enrollment and initiation of treatment.  Muscle biopsy will be obtained whenever possible as part of the standard of care.
  • Subjects must be willing and able to provide written informed consent.

Contact Info:

Research Coordinator, Leslie Lazar Thorn, 206-744-9143 / 206-295-4425, lazthorn@uw.edu