Gullian Barre Syndrome Clinical Trial
Official title:
Inhibition of Complement Activation (Eculizumab) in Guillain-Barre Syndrome Study
Guillian-Barre Syndrome (GBS) is the most frequent cause of acute neuromuscular weakness in
the Western World and can occur at any age. GBS is a rpadily progressive 'inflammatory'
disorder of the perihperal nerves often leading to sever paresis of the limbs. Most GBS
patients also have sensory disturbances (tingling or dull feeling) and pain. Some patients
also have double vision or problems with swallowing. GBS mau also involve the respiratory
muscles, leading to insufficient ventilation and admission to an intensive care unit. GBS
pateints have a vairable prognosis; 20-30% require mechnical ventilation for a period
ranging from weeks to months, 20% are unable to walk after 6 months nad 3-5% dies.
Progression of weakness in GBS is usually rapid and reaches its peak within 4 weeks in the
majority of patients, but many develop their maximum deficit within 2 weeks. Thereafter, the
patients have a variable prognosis.
GBS is a treatable disorder. Intravenous immunoglobulin (IVIg) 2g/kg administered in 5 days
was shown to be effective when administered within the first two weeks after onset of
symptoms, and is considered the treatment of choice by most experts in the field. Although
the standard treatment for GBS is a single course of IVIg (2g/kg administered in 5 days),
many patients fails to recover abd remain with substantial disability. Patients with GBS and
especially those with a poor prognosis potentially may benefit from more powerful abd when
possible a more mechanistically rational therapy.
Recent experimental evidence suggests that complement activation palys a crucial role in the
development of neuromuscular weakness in GBS making complement inhibitors and regulators
attracive therapeutic targets. Our hypothesis is that Eculizumab, with its function as a
complement inhibitor, will be very effective in preventing progression of weakness in
patients with GBS.
n/a
Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment