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.
Study Type
OBSERVATIONAL
Enrollment
22
Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase
A >50% change from baseline on the Stiffness Index scores ( scale from 0-6; each item adds one) after 12 weeks of treatment.
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
Time frame: 24 MONTHS
A >50% change from baseline on the Heightened Sensitivity scores (scales from 1-7, each item adds one) after 12 weeks of treatment.
Proving that SCIg is as effective as IVIg on this clinical measure will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
Time frame: 24 months
A meaningful change on Quality of Life (QoL) measures after 12 weeks of SCIg based on 6 sets of QoL Questionnaires (mobility, self-care, usual activities, pain, anxiety/depression, health state)
Proving that SCIg affects Quality of life, as IVIg does, will be important for the patients who may have another treatment option avoiding the systemic side effects of IVIg
Time frame: 24 MONTHS
If SCIg reduces the anti-GAD antibody titers measured in the patient's blood samples by more than 30%
An effect on the circulating anti-Glutamic Acid Decarboxylase (GAD) antibodies will elucidate the mechanism of action of SCIg and any correlation between efficacy and antibody titers
Time frame: 24 MONTHS
If >50% of patients prefer either SCIg or IVIg after 12 weeks of treatment based on a YES/NO questionnaire .
Proving patients' preference is important because SCIg is self-administered at home and more convenient, without the need for hospitalizations.
Time frame: 24 MONTHS
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