Monotherapy IVIG Gamunex-C for HMG-CoA Reductase Auto-Antibody Positive Necrotizing Myopathy Treatment (The MIGHT Trial)

  • STATUS
    Recruiting
  • days left to enroll
    19
  • participants needed
    10
  • sponsor
    University of Washington
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Updated on 24 March 2022
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Summary

This is a phase 2, pilot, randomized, placebo-controlled trial of Gamunex-C IVIG as mono-therapy for HMGCoA reductase auto-antibody positive (HMGCR) necrotizing myopathy. The trial will test the feasibility and initial efficacy of Gamunex-C IVIG mono-therapy in HMGCR necrotizing myopathy.

Description

This is a phase 2, double-blinded, randomized, placebo-controlled, multi-center trial of Gamunex-C IVIG as mono-therapy for HMGCR necrotizing myopathy. Up to 10 treatment-naïve patients will be enrolled and randomized to receive either Gamunex-C IVIG dosed at 2g/kg or placebo at week 0 and week 4. The primary efficacy outcome is the percentage of patients at week 8 with at least minimal improvement per the 2016 ACR/EULAR myositis clinical response criteria.

Details
Condition Immune-Mediated Necrotizing Myopathy
Treatment Albumin, GAMUNEX-C
Clinical Study IdentifierNCT04450654
SponsorUniversity of Washington
Last Modified on24 March 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

A subject must meet all of the following inclusion criteria at screening to be eligible for
participation in this study
Anti-HMGCR positive. Patients will be screened by commercially-available ELISA
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)
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
Subjects must be willing and able to provide written informed consent
enrollment and initiation of treatment. Muscle biopsy will be obtained whenever
possible as part of the standard of care

Exclusion Criteria

Disease duration greater than 36 months
A subject meeting any of the following exclusion criteria at screening is NOT eligible for
participation in this study
Exposure to immunoglobulin treatment (IV, IM, or SubQ) in the prior 3 months
Participants taking oral or intravenous glucocorticoids where the dose has changed
Exposure to plasma exchange (PEX) in the prior 3 months
within 4 weeks of screening
Exposure to rituximab or any monoclonal antibody in the prior 12 months
Exposure to other immunosuppressive medications (e.g. methotrexate, leflunomide
Presence of respiratory or swallowing dysfunction due to HMGCR myopathy
azathioprine, mycophenolate mofetil) in the prior 6 months
Inadequate venous access
History of anaphylactic reactions or severe reactions to any blood-derived product
Currently taking a statin medication
History of dermatomyositis rash (either biopsy-proven, or history of photosensitive
History of intolerance to any component of the IP
rash)
History of thrombotic complication to polyclonal IVIG therapy
History of pulmonary embolism or deep venous thromboembolism
History of hyperviscosity or hypercoagulable state
History of myocardial infarction or stroke in the last 12 months
History of chronic liver disease
Currently receiving anti-coagulation therapy (vitamin K antagonists, non-vitamin K
oral anticoagulants [e.g. dabigatran, rivaroxaban, apixaban], parenteral
Hemoglobin level <9 g/dL
anticoagulants [e.g fondaparinux]. Note that oral anti-platelet agents are allowed
Known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies
(e.g. aspirin, clopidogrel, ticodipine)
History of chronic alcoholism or illicit drug abuse (addiction) in the prior 12 months
Females of child-bearing potential who are pregnant, have a positive serum pregnancy
test (human chorionic gonadotropin [HCG]-based assay), breastfeeding, or are unwilling
to practice a highly effective method of contraception (oral, injectable or implanted
hormonal methods of contraception, placement of an intrauterine device or intrauterine
History of angina pectoris or transient ischemic attack (TIA) in the last 12 months
system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male
sterilization, or true abstinence) throughout the study
Wells Criteria Score for DVT of 2 or more at the time of screening
Wells Criteria Score for PE of 4 or more at the time of screening
True abstinence: When this is in line with the preferred and usual lifestyle of the
subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal
post-ovulation methods], declaration of abstinence for the duration of a trial, and
withdrawal are not acceptable methods of contraception.)
Renal impairment (i.e., estimated glomerular filtration rate (eGFR) below 60ml/min)
Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) levels exceeding
more than 2.5 times the ULN for the expected normal range for the testing laboratory
not due to HMGCR myopathy
Active psychiatric illness that interferes with compliance or communication with
healthcare personnel
Currently receiving, or having received, within 1 month prior any investigational
medicinal product or device. In the case of an investigational medicinal product
trial, at least five half- lives (if known) must have elapsed prior to Screening
Any medical condition which makes the clinical trial participation unadvisable or
which is likely to interfere with the evaluation of the study treatment and/or the
satisfactory conduct of the clinical trial according to the investigator's judgment
Any factor that in the opinion of the investigator would compromise the ability of the
subject to complete the trial
Weight > 120kg. Individuals weighing >100kg and ≤120kg will be eligible at the
discretion of the investigators
Presence of a central, in-dwelling catheter (such as a PICC line) at the time of
informed consent
Currently taking a nephrotoxic drug (eg gentamicin or vancomycin) at the time of
informed consent
Severe cardiac failure at the time of informed consent
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