| Eligibility |
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.
- Participants taking oral or intravenous glucocorticoids where the dose has changed
within 4 weeks of screening.
- 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
- Currently taking a statin medication
- 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., estimated glomerular filtration rate (eGFR) below 60ml/min)
- 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.
- 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.
|
