Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome (SPS)

Study Design:This is a proof of concept observational prospective, open label, study on the safety, efficacy and convenience of treatment with SCIg study of 22 patients at Thomas Jefferson University Hospital. Two cohorts of patients within the total of 22 will be included; half of them (11 patients) currently receiving and responding to IVIg and the other half starting de novo on SCIg. Patients diagnosed with SPS according to defined sets of symptoms will be eligible to enroll. The primary clinical outcome will be based on clinical efficacy measures, as used before for the IVIg trial, based on changes in the Stiffness Index and Heightened Sensitivity scores, using the validated scales that the investigators have had previously utilized and validated (Dalakas et al 2001; see attached at the end of the protocol). These same measurements will be applied while on IVIg (weeks 0, 4, 8, 12) and will be compared to the measurements obtained during SCIg (weeks 16, 20, 24, 28). The secondary outcome will be Quality of Life (QoL) responses and patient preference for each treatment.