Clinical trial NCT06703333: Extracorporeal Photopheresis (ECP) in the Treatment of Stiff Person Syndrome (OPTION Study)
Official title: A Pilot Study Evaluating the Safety of Extracorporeal Photopheresis (ECP) in the Treatment of Stiff Person Syndrome (OPTION Study)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: EARLY_PHASE1
Conditions: Stiff Person Syndrome
Keywords: Stiff Person Syndrome
Extracorporeal Photopheresis
ECP
Immunoregulation
Lead sponsor: Abu Dhabi Stem Cells Center
Sponsor class: OTHER_GOV
First posted: 2024-11-25
Enrollment: 3
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Tolerability to ECP procedures
timeFrame: Weeks 0-24
measure: Incidence of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) assessed by CTCAE v5.0.
timeFrame: Weeks 0-52
measure: Tolerability to TEAEs, AESIs, and SAEs
timeFrame: Weeks 0-52
Secondary outcomes:
measure: Proportion of subjects with clinical improvement
timeFrame: Baseline, months 3, 6, 9, and 12
Other outcomes:
Locations:
city: Abu Dhabi
country: United Arab Emirates
facility: Abu Dhabi Stem Cells Center
Study officials:
References:
citation: 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.