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

October 1, 2025 updated by: Yandy Marx Castillo Aleman

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

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
3

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Tolerability to ECP procedures
Time Frame: Weeks 0-24
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.
Time Frame: Weeks 0-52
Proportion of patients referring TEAEs, AESIs, and SAEs.
Weeks 0-52
Tolerability to TEAEs, AESIs, and SAEs
Time Frame: Weeks 0-52
Proportion of patients tolerating TEAEs, AESIs, and SAEs, and finalizing the planned study.
Weeks 0-52

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Proportion of subjects with clinical improvement
Time Frame: Baseline, months 3, 6, 9, and 12

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.
Baseline, months 3, 6, 9, and 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Yandy Marx Castillo Aleman

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 21, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 21, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 25, 2024

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 6, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 1, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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