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

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

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Stiff Person Syndrome
• Intervention / Treatment: Combination product: Extracorporeal photopheresis (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).

• Study Type: Interventional
• Enrollment (Estimated): 3
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Yandy M. Castillo Aleman, MD; Phone Number: +971800810; Email: yandy.castillo@adscc.ae
• Study Contact Backup: Name: Pierre C. Krystkowiak, MD, PhD; Email: pierre.krystkowiak@capital-health.ae

Study Locations

• United Arab Emirates
  • Abu Dhabi, United Arab Emirates, 4600
    • Abu Dhabi Stem Cells Center
    • Contact: Yandy M. Castillo Aleman, MD; Phone Number: +971800810; Email: yandy.castillo@adscc.ae
    • Contact: Ruqqia Mir, MBBS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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).
• Platelet count ≥ 100 x 10^3/uL (with or without transfusion support).
• Willingness to use at least one reliable method of birth control (e.g., abstinence, oral contraceptives, intrauterine devices, barrier method with spermicide, or surgical sterilization) throughout the study for all men and women of childbearing potential.
• Willingness to participate in all study tests, visits, and procedures (including ECP), as outlined in the informed consent.
• Adequate peripheral venous access to initiate ECP therapy.
• The patient agrees to participate in the trial and signs the informed consent form.

Exclusion Criteria:

• 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.

• Absolute medical contraindication to receive ECP.

  • Hypersensitivity or allergy to psoralen (methoxalen).
  • Hypersensitivity or allergy to heparin.
  • Previous history of heparin-induced thrombocytopenia.
  • Aphakia or photosensitive disease (systemic lupus erythematosus, porphyrias, etc.).

• Laboratory evidence of any of the following:

  • Mononuclear cell (MNC) count <2.0 x 10^3 cells/uL.
  • Serum transaminase levels > x 2 UNL.
  • Estimated Glomerular Filtration Rate (eGFR) <60 mL/min/1.73 m2.
• Evidence of known infection with human immunodeficiency virus (HIV) or hepatitis B.
• Uncontrolled infection requiring treatment at study entry.
• Use of any investigational drug/treatment at the time of enrollment or within the previous 60 days, or five elimination half-lives, or until the expected pharmacodynamic effect has returned to baseline, whichever is longer.
• Previous history of skin cancer, leukemia / lymphoma / myeloma, or bone marrow transplant.
• Any other disease or condition which, in the opinion of the investigator, could interfere with participation according to the study Protocol, or with the ability of the patients to cooperate and comply with study procedures.
• Inability to provide informed consent.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental arm; Single group assignment in which SPS patients will receive ECP as an add-on therapy on the following schedule: 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).	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).; Other Names: Extracorporeal photochemotherapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tolerability to ECP procedures	Proportion of patients tolerating the ECP procedures reaching the cycles' goal.	Weeks 0-24
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.	Weeks 0-52
Tolerability to TEAEs, AESIs, and SAEs	Proportion of patients tolerating TEAEs, AESIs, and SAEs, and finalizing the planned study.	Weeks 0-52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.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.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.	Baseline, months 3, 6, 9, and 12

Collaborators and Investigators

• Sponsor: Yandy Marx Castillo Aleman

Publications and helpful links

General Publications

• Castillo-Aleman YM, Krystkowiak PC. Extracorporeal photopheresis in stiff person syndrome. Front Immunol. 2024 Dec 16;15:1519032. doi: 10.3389/fimmu.2024.1519032. eCollection 2024. No abstract available.

Study record dates

Study Major Dates

• Study Start (Estimated): January 1, 2026
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): July 1, 2027

Study Registration Dates

• First Submitted: November 21, 2024
• First Submitted That Met QC Criteria: November 21, 2024
• First Posted (Actual): November 25, 2024

Study Record Updates

• Last Update Posted (Estimated): October 6, 2025
• Last Update Submitted That Met QC Criteria: October 1, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: ECP; Extracorporeal Photopheresis; Immunoregulation; Stiff Person Syndrome
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Spinal Cord Diseases; Stiff-Person Syndrome; Therapeutics; Surgical Procedures, Operative; Phototherapy; Extracorporeal Circulation; PUVA Therapy; Ultraviolet Therapy; Photopheresis
• Other Study ID Numbers: CT.009.1.0.OPTION; MF-5527-2024-19 (Other Identifier: ADSCC Institutional Review Board (IRB))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: Yes