NCT06703333

Extracorporeal Photopheresis (ECP) in the Treatment of Stiff Person Syndrome (OPTION Study)

A Pilot Study Evaluating the Safety of Extracorporeal Photopheresis (ECP) in the Treatment of Stiff Person Syndrome (OPTION Study)

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 3 (estimated)
• Sponsor: Yandy Marx Castillo Aleman · Other Government
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

The study complies with the current Good Clinical Practices (GCPs) and follows the principles of the Declaration of Helsinki. The primary objective is the safety profile assessment of the investigational intervention, to be assessed by ECP procedure tolerability, the incidence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) according to the Common Terminology Criteria for Adverse Events (CTCAE v5.0), and the World Health Organization - Uppsala Monitoring Center (WHO-UMC) causality assessment system. The ECP preliminary efficacy assessment, as a secondary objective, will be assessed by the 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).

Conditions

• Stiff Person Syndrome

Interventions

Type	Name	Description
COMBINATION_PRODUCT	Extracorporeal photopheresis (ECP)	* 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).

Timeline

• Start date: 2026-01-01
• Primary completion: 2027-04-01
• Completion: 2027-07-01
• First posted: 2024-11-25
• Last updated: 2025-10-06

Locations

1 site across 1 country: United Arab Emirates

Regulatory

• FDA-regulated drug study
• FDA-regulated device study