Intravenousimmunoglobulin (IVIg) for the Treatment of Inflammatory Myopathies
Information source: National Institutes of Health Clinical Center (CC)
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Dermatomyositis; Inclusion Body Myositis; Polymyositis
Intervention: Gamma Globulin (Drug)
Phase: Phase 2
Sponsored by: National Institute of Neurological Disorders and Stroke (NINDS)
Inflammatory myopathies are a group of muscle diseases characterized by muscle weakness, high
levels of muscle enzymes in the blood, and inflammation of the tissue surrounding muscle
The diseases making up the inflammatory myopathies are grouped into three subsets:
I) Polymyositis (PM)
II) Dermatomyositis (DM)
III) Inclusion Body Myositis (IBM)
Inflammatory myopathies are thought to be autoimmune processes and are treated with steroids
and immunosuppressive drugs. However, many patients who initially respond to these
treatments develop resistance to the therapy or experience side effects causing the
treatments to be stopped.
Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with PM, DM,
and IBM a safer and more effective alternative to standard therapies for the diseases. IVIg
is a drug that has been used successfully to treat other immune-related diseases of the
The study will take 60 patients and divide them into two groups. Group one will receive 2
injections of IVIg once a month for three months. Group two will receive 2 injections of
placebo "inactive injection of sterile water" once a month for three months. Following the
three months of treatment, group one will begin taking the placebo and group two will begin
taking IVIg for an additional 3 months. The drug will be considered effective if patients
receiving it experience a significant improvement (>15%) in muscle strength.
Official title: The Efficacy of High-Dose Intravenous Immunoglobulin in Patients With Inflammatory Myopathies: A Three Month Randomized Trial With Option for Cross-Over
Study design: Treatment, Safety/Efficacy Study
The inflammatory myopathies are a group of acquired muscle diseases characterized by subacute
onset of progressive proximal muscle weakness, elevated serum muscle enzymes and endomysial
inflammation. They comprise 3 clinically distinct subsets: polymyositis (PM),
dermatomyositis (DM) and Inclusion Body Myositis (IBM). Because immune-mediated mechanisms
are primarily responsible for the clinical manifestations of these conditions, the treatment
of choice is with corticosteroids or immunotherapy drugs. Although most of the patients
initially respond to these drugs, a number of them become resistant or develop unacceptable
side effects that necessitate their discontinuation. The need for a more effective and safe
immunotherapy in patients with PM, DM or IBM prompted the present study using high dose
intravenous immunoglobulin (IVIg). IVIg is an immunomodulating agent which has been shown to
be effective and safe in the treatment of a number of patients with immune-related
This is a double-blind, randomized, placebo-controlled study involving 30 patients, who will
receive IVIg or placebo for 3 months and then will cross-over to the alternate therapy for
another period of 3 monthly infusions. The monthly dose of IVIg is 2 GM/Kg divided into two
daily doses. The drug will be considered effective if patients experience an increase of
more than 15% in their baseline muscle strength. Muscle strength will be assessed with a
series of objective dynamometric measurements performed before and at the end of each monthly
Minimum age: N/A.
Maximum age: N/A.
Selected patients should have PM, IBM or DM.
Specifically they should have a) proximal muscle weakness; b) no evidence of clinical,
histological or family history of another neuromuscular illness; c) elevation of muscle
enzymes during the course of the disease; d) typical skin rash in case of DM; and e)
diagnostic muscle biopsy.
Suitable candidates for IVIg should be patients with active, bonefide disease who:
1. have been treated with steroids but had: a) no response or incomplete response (as
defined by continued muscle weakness) to high-dose therapy or b) a good response to
steroids but inability to taper the dose without a flare of disease activity or c)
unacceptable steroid side effects such as gastrointestinal hemorrhages, osteonecrosis,
hyperglycemia, extreme weight gain etc., and
2. have been treated with one immunosuppressive drug (such as azathioprine, Methotrexate,
Cyclophosphamide, Cyclosporine) but without benefit or with unacceptable side
Pregnant or nursing women (confirmed by a screening pregnancy test).
Critically ill patients such as those requiring intravenous pressors for maintenance of
cardiac output due to severe cardiomyopathy, patients with respiratory insufficiency and
patients with severe muscle weakness requiring help for basic self care.
Children below age 18.
Patients with severe renal or hepatic disease, severe COPD or coronary artery disease or
other systemic medical problems often seen when PM or DM is associated with severe cases of
lupus, rheumatoid arthritis or scleroderma.
Patients with known allergic reaction to IVIg.
Serum IgA less than 11mg/dl.
Locations and Contacts
National Institute of Neurological Disorders and Stroke (NINDS), Bethesda, Maryland 20892, United States
Dalakas MC. Polymyositis, dermatomyositis and inclusion-body myositis. N Engl J Med. 1991 Nov 21;325(21):1487-98. Review. No abstract available.
Dalakas M. Treatment of polymyositis and dermatomyositis. Curr Opin Rheumatol. 1989 Dec;1(4):443-9. Review. No abstract available.
Roifman CM, Schaffer FM, Wachsmuth SE, Murphy G, Gelfand EW. Reversal of chronic polymyositis following intravenous immune serum globulin therapy. JAMA. 1987 Jul 24-31;258(4):513-5. No abstract available.
Starting date: May 1990
Ending date: July 2002
Last updated: March 3, 2008