Intravenous Immunoglobulin (IVIg) for the Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Information source: National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Demyelinating Diseases; Paraproteinemias
Intervention: intravenous immunoglobulin (IVIg) (Drug)
Phase: Phase 2
Sponsored by: National Institute of Neurological Disorders and Stroke (NINDS)
Chronic Inflammatory Demylinating Polyneuropathy (CIDP) is an autoimmune condition affecting
the nervous system. Researchers believe the immune system begins attacking the cells
covering nerves called myelin. The destruction of myelin causes muscle weakness, loss of
sensation, abnormal levels of protein in the fluid surrounding the brain (CSF), and slowing
of the nervous system. The disease progresses slowly and disables patients suffering from
CIDP is treated with steroids, plasmapheresis, and immunosuppressive drugs. Many patients
initially respond to these treatments, but 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 CIDP a
safer and more effective alternative to standard therapies for the disease. IVIg is a drug
that has been used successfully to treat other immune-related diseases of the nervous
system. However, because IVIg is so expensive, researchers believe it should first be
proven effective on a small group of patients.
The study will take 60 patients with CIDP 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 (>25%) in muscle strength.
Official title: The Efficacy of High-Dose Intravenous Immunoglobulin in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Study design: Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a slowly progressive disabling
neuropathy characterized by subacute onset of muscle weakness, distal sensory deficit,
elevated spinal fluid protein, and slow nerve conduction velocity with or without conduction
block. A monoclonal gammopathy is at times present in the serum of some patients. Because
immune-mediated mechanisms against peripheral nerve myelin are thought to be primarily
responsible for the clinical manifestations of CIDP, the treatment of choice is with
corticosteroids, plasmapheresis or immunosuppressive drugs. Although many patients
initially respond to these agents, a large number of them become resistant or develop
unacceptable side effects that necessitate their discontinuation. The need for a more
effective and safe immunotherapy in CIDP patients prompted the present study using high-dose
intravenous immunoglobulin (IVIg). IVIg is an immunomodulating agent which has been
recently shown to be effective and safe in the treatment of a number of patients with
immune-related neuromuscular diseases.
This is a double blind, randomized, placebo controlled, trial involving 60 patients, half of
which will receive IVIg and the other half placebo (D5/W). Because IVIg is prohibitively
expensive, a controlled trial is needed to provide convincing evidence of efficacy, and
ensure that the benefit is not due to spontaneous improvement or to observer bias. The dose
of IVIg is 2 GM/Kg divided into two daily doses administered monthly for six months. The
drug will be considered effective if patients experience an increase of more than 25% in
their baseline muscle strength. Muscle strength will be assessed with a series of objective
dynamometric measurements performed before and after each monthly infusion.
Minimum age: N/A.
Maximum age: N/A.
Selected patients should have CIDP with or without an associated monoclonal gammopathy.
Subjects should have clinical evidence of peripheral neuropathy with muscle weakness and
Subjects should have evidence of clinical, histological or family history of another
Subjects should have elevation of CSF protein during the course of the disease.
Subjects should have demyelination by nerve conduction study and/or nerve biopsy.
Suitable candidates for IVIg should be patients with active, bonefide CIDP 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. or
2. have been additionally treated with one of the other immunosuppressive agents
considered effective in some CIDP patients, such as azathioprine, chlorambucil,
cyclophosphamide, cyclosporine or plasmapheresis but without benefit or with
unacceptable side effects that had necessitated their discontinuation.
Subjects should not be pregnant or nursing.
Subjects should not be critically ill such as those requiring intravenous pressors for
maintenance of cardiac output, patients with unstable respiratory insufficiency and
patients with such severe muscle weakness requiring help for basic self care (Karnofsky
performance scale less than 50).
No subjects below 18 years of age.
Patients should not have severe renal or hepatic disease and severe COPD or coronary
Patients should not be allergic to IVIg or have a known IgA deficiency.
Locations and Contacts
National Institute of Neurological Disorders and Stroke (NINDS), Bethesda, Maryland 20892, United States
Dalakas MC, Engel WK. Chronic relapsing (dysimmune) polyneuropathy: pathogenesis and treatment. Ann Neurol. 1981;9 Suppl:134-45.
Cook D, Dalakas M, Galdi A, Biondi D, Porter H. High-dose intravenous immunoglobulin in the treatment of demyelinating neuropathy associated with monoclonal gammopathy. Neurology. 1990 Feb;40(2):212-4.
Basta M, Dalakas MC. High-dose intravenous immunoglobulin exerts its beneficial effect in patients with dermatomyositis by blocking endomysial deposition of activated complement fragments. J Clin Invest. 1994 Nov;94(5):1729-35.
Starting date: December 1990
Last updated: March 3, 2008