The purpose of this study is to evaluate the efficacy and safety of QIVIGY (Intravenous Human Normal Immune Globulin 10%) compared with placebo in adult participants with stiff person syndrome (SPS).
This is a phase III, double-blind, placebo-controlled, parallel group, multicenter study to assess the efficacy and safety of QIVIGY in adult participants with stiff person syndrome. Participants will be randomized in a 1:1 ratio, to receive either 2 g/kg of QIVIGY or equivalent volume of placebo. During the randomized phase, study treatment will be administered every 4 weeks for 24 weeks, at which point the primary efficacy endpoint assessment will be performed at an End of Randomized Treatment visit. At this study visit, all participants will be given the option to enter an open-label study extension. Participants who choose to continue in the study will receive 2.0 g/kg QIVIGY every 4 weeks, for a duration of 24 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
38
Intravenous human normal immunoglobulin (IVIg) 10%
Human albumin solution, diluted with saline 0.9% to a final concentration of 0.5% as an intravenous infusion
Timed 25-Foot Walk (T25-FW)
Proportion of participants showing an improvement on the Timed 25-Foot Walk
Time frame: Baseline to Week 24
Timed 25-Foot Walk (T25-FW)
Change in the Timed 25-Foot Walk from baseline to Week 24 and, for participants entering the study extension, to Week 48
Time frame: Baseline up to Week 48
Distribution-of-Stiffness (DSI)
Change in the scores of the distribution-of-stiffness index (DSI) from baseline to Week 24 and, for participants entering the study extension, to Week 48
Time frame: Baseline up to Week 48
Heightened Sensitivity Scale (HSS)
Change in heightened sensitivity scale (HSS) from baseline to Week 24 and, for participants entering the study extension, to Week 48
Time frame: Baseline up to Week 48
Modified Rankin Scale (mRS)
Change in Modified Rankin scale (mRS) from baseline to Week 24 and, for participants entering the study extension, to Week 48
Time frame: Baseline up to Week 48
HRQoL assessments (Neuro-QOL)
Change in score by visit
Time frame: Baseline up to Week 48
Pain Interference Score
Change in score by visit
Time frame: Baseline up to Week 48
Change in level of walking aid dependency
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Change from baseline in level of walking aid dependency will be assessed by clinician-reported ambulatory status during scheduled clinical outcome assessments. Walking aid use (unaided, cane, or walker) will be recorded at baseline and at each study visit. Improvement is defined as a reduction in level of assistance (e.g., from walker to cane or unaided, or from cane to unaided).
Time frame: Baseline up to Week 48
Assess Safety
Assess treatment-emergent AEs and SAEs
Time frame: Day 0 to Week 48
Duration of Response
Participants on QIVIGY in the randomized phase who responded to treatment will be assessed for continued change (duration of response) through 12 months, based on the T25-FW Test
Time frame: Week 24 to Week 48
Time to response
Participants on placebo in the randomized phase and did not achieve a response will be assessed for time to response to QIVIGY based on the T25-FW Test
Time frame: Week 24 to Week 48