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

NCT06703333 | Not Yet Recruiting Early Phase 1 DMC FDA Drug FDA Device

• 2 other identifiers: CT.009.1.0.OPTIONMF-5527-2024-19
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Stiff Person Syndrome

Keywords

Stiff Person Syndrome; Extracorporeal Photopheresis; ECP; Immunoregulation

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

• Proportion of subjects with clinical improvement

  Baseline, months 3, 6, 9, and 12

Study Arms (1)

Experimental arm

EXPERIMENTAL

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)

Interventions

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).

Also known as: Extracorporeal photochemotherapy

Experimental arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Yandy Marx Castillo Aleman: lead

Study Sites (1)

Abu Dhabi Stem Cells Center

Abu Dhabi, 4600, United Arab Emirates

Location

Related Publications (1)

• 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.

  PMID: 39759515 BACKGROUND

MeSH Terms

Conditions

Stiff-Person Syndrome

Interventions

Photopheresis

Condition Hierarchy (Ancestors)

Autoimmune Diseases of the Nervous System; Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

PUVA Therapy; Ultraviolet Therapy; Phototherapy; Therapeutics; Extracorporeal Circulation; Surgical Procedures, Operative

Central Study Contacts

Yandy M. Castillo Aleman, MD

CONTACT

+971800810; yandy.castillo@adscc.ae

Pierre C. Krystkowiak, MD, PhD

CONTACT

pierre.krystkowiak@capital-health.ae

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Specialist Immunologist

Study Record Dates

• First Submitted: November 21, 2024
• First Posted: November 25, 2024
• Study Start: January 1, 2026
• Primary Completion (Estimated): April 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: October 6, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

AE (1)