OPTION study is a pilot, open-label, prospective, and multicentric clinical trial involving outpatients with a diagnosis of classical stiff person syndrome (SPS), whereas extracorporeal photopheresis (ECP) is the investigational treatment. The study will be conducted at the Specialized Rehabilitation Hospital/Capital Health and Yas Clinic Khalifa City (YCKC) Hospital (managed by Abu Dhabi Stem Cells Center -ADSCC), clinical sites responsible for patient assessment and inclusion, and follow-up consultations, according to the approved Protocol, while YCKC will be the site in which patients will undergo the investigational intervention (ECP).
Documented history of classical SPS with serum and CSF anti-GAD antibodies.
Age ≥ 18 ≤ 75 years.
Weight ≥ 50 kg.
Hematocrit ≥ 30 % (with or without transfusion support).
Progressive encephalomyelitis with rigidity and myoclonus (PERM).
Paraneoplastic variants of SPS.
Concurrent diagnosis of a neurological condition that would interfere with the assessment of SPS.
Women who are pregnant and/or lactating.
Single group
Extracorporeal photopheresis (ECP)
Combination product* Months 1-3: One ECP cycle (two-consecutive days) every other week for 3 months (12 procedures). * Months 4-6: One ECP cycle every month for 3 months (6 procedures). * Total: 18 procedures (within 6 months \~24 weeks).
Tolerability to ECP procedures
Proportion of patients tolerating the ECP procedures reaching the cycles' goal.
Incidence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) assessed by CTCAE v5.0.
Proportion of patients referring TEAEs, AESIs, and SAEs.
Tolerability to TEAEs, AESIs, and SAEs
Proportion of patients tolerating TEAEs, AESIs, and SAEs, and finalizing the planned study.
Proportion of subjects with clinical improvement
Proportion of SPS subjects who are treatment responders, defined as: * ≥ 2 points reduction in the Distribution of Stiffness Index (DSI), and * ≥ 1 point reduction in Heightened Sensitivity Score (HSS). Marker for efficacy of the investigational intervention. 1. DSI: Scores on this index range from 0 to 6 and reflect the extent of stiffness; with one point being given for stiffness in each of the following areas: lower trunk, upper trunk, legs, arms, face, and abdomen. Lower scores indicate less stiffness. 2. HSS: Scores range from 1 to 7 based on response; one point being given for each source of or type of spasm, as follows: unexpected noises, visual stimuli, somatosensory stimuli, voluntary activities, emotional upset or stress, no specific stimuli, and nocturnal spasms. Lower scores indicate less frequent spasms.
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Yandy Marx Castillo Aleman
Lead sponsor
Abu Dhabi Stem Cells Center
Sponsor institution
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