Stiff-Person Syndrome Clinical Trial
Official title:
Efficacy and Mechanism of Action of SCIg in Patients With Stiff Person Syndrome
This is a pilot, proof-of concept investigator-initiated trial planned for 22 patients with the diagnosis of Stiff Person Syndrome (SPS). The study will compare efficacy of treatment using subcutaneous immunoglobulin therapy (SCIg) compared to intravenous immunoglobulin (IVIg) therapy. The majority of IVIg naïve subjects (those not already receiving IVIg) are typically managed with non-immunotherapy mostly Gamma Aminobutyric Acid (GABA) -enhancing drugs such as Baclofen or Diazepam.
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.
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