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

  • STATUS
    Recruiting
  • End date
    Jul 20, 2022
  • participants needed
    10
  • sponsor
    University of Washington
Updated on 20 September 2021

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-nave 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 on20 September 2021

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 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

Exclusion Criteria

A subject meeting any of the following exclusion criteria at screening is NOT
eligible for participation in this study
Disease duration greater than 36 months
Currently taking glucocorticoids
Participants will need to be tapered off all glucocorticoids by their treating physician over an interval deemed appropriate, and be free of steroid use for at least 14 days prior to the time of study screening in order to be eligible
Exposure to immunoglobulin treatment (IV, IM, or SubQ) in the prior 3 months
Exposure to plasma exchange (PEX) in the prior 3 months
Exposure to other immunosuppressive medications (e.g. methotrexate, leflunomide, azathioprine, mycophenolate mofetil) in the prior 6 months
Exposure to rituximab or any monoclonal antibody in the prior 12 months
Exposure to a statin in the prior 3 months
History of dermatomyositis rash (either biopsy-proven, or history of photosensitive rash)
Presence of respiratory or swallowing dysfunction due to HMGCR myopathy
Inadequate venous access
History of anaphylactic reactions or severe reactions to any blood-derived product
History of intolerance to any component of the IP
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
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, ticodipine)
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 system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence) throughout the study
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., serum creatinine exceeds more than 1.5 times the upper limit of normal [ULN] for the expected normal range for the testing laboratory)
History of chronic liver disease
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
Hemoglobin level <9 g/dL
Known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies
History of chronic alcoholism or illicit drug abuse (addiction) in the prior 12 months
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
History of angina pectoris or transient ischemic attack (TIA) in the last 12 months
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
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