Stiff Person SyndromeEarly Phase 1OPTIONNCT06703333
Extracorporeal Photopheresis (ECP) in the Treatment of Stiff Person Syndrome (OPTION Study)
A Early Phase 1 study testing Extracorporeal photopheresis (ECP) in people with Stiff Person Syndrome, run by Yandy Marx Castillo Aleman at 1 site in United Arab Emirates.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Stiff Person Syndrome
- What is being tested?
- Extracorporeal photopheresis (ECP)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited Arab Emirates
- Study dates
- Aug 2026to Jul 2027
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
1 site in 1 country.
United Arab Emirates1 site
Abu Dhabi Stem Cells Center
Abu Dhabi, 4600
About this study
Official title: 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).
Read the 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
- Being tested
- Extracorporeal photopheresis (ECP)
- Keywords
- Stiff Person Syndrome, Extracorporeal Photopheresis, ECP, Immunoregulation
- Medical subject headings
- Stiff-Person Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Extracorporeal photopheresis (ECP)Combination product
Also called Extracorporeal photochemotherapy
- 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).
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Combination Product: Extracorporeal photopheresis (ECP) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tolerability to ECP procedures
Proportion of patients tolerating the ECP procedures reaching the cycles' goal.
Measured: 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.
Measured: Weeks 0-52
Tolerability to TEAEs, AESIs, and SAEs
Proportion of patients tolerating TEAEs, AESIs, and SAEs, and finalizing the planned study.
Measured: Weeks 0-52
Secondary outcomes (1)
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.
Measured: Baseline, months 3, 6, 9, and 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2024
- Study startAug 2026 (estimated)
- Primary completionApr 2027 (estimated)
- Study completionJul 2027 (estimated)
Why it stopped: Unavailability of funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Stiff Person SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yandy Marx Castillo Aleman
- Sponsor type
- Other government
- Responsible party
- Sponsor-investigator, Yandy Marx Castillo Aleman, Abu Dhabi Stem Cells Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06703333
- Sponsor's protocol ID
- CT.009.1.0.OPTION
- Other IDs
- MF-5527-2024-19
- Acronym
- OPTION
- Status last verified
- Aug 2026
- First submitted
- 21 Nov 2024
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
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.
PubMed 39759515
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study Evaluating the Safety of Extracorporeal Photopheresis (ECP) in the Treatment of Stiff Person Syndrome (OPTION Study)
- ClinicalTrials.gov ID
- NCT06703333
- Acronym / sponsor ID
- OPTION / CT.009.1.0.OPTION
- Conditions
- Stiff Person Syndrome
- Keywords
- Stiff Person Syndrome, Extracorporeal Photopheresis, ECP, Immunoregulation
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Unavailability of funding
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Yandy Marx Castillo AlemanOther government
- Study start
- Aug 2026 (estimated)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 25 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental arm | Experimental | Combination Product: Extracorporeal photopheresis (ECP)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). |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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).
Key exclusion criteria (19 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Extracorporeal photopheresis (ECP)Also: Extracorporeal photochemotherapy* 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). | Combination product | Experimental arm |
Primary outcome
Tolerability to ECP procedures
Time frame: Weeks 0-24
Proportion of patients tolerating the ECP procedures reaching the cycles' goal.
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.
Tolerability to TEAEs, AESIs, and SAEs
Time frame: Weeks 0-52
Proportion of patients tolerating TEAEs, AESIs, and SAEs, and finalizing the planned study.
1 secondary outcome
Proportion of subjects with clinical improvement
Time frame: Baseline, months 3, 6, 9, and 12
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2026.
- Results publication: PubMed 39759515Castillo-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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Extracorporeal photopheresis (ECP) in people with Stiff Person Syndrome, run by Yandy Marx Castillo Aleman at 1 site in United Arab Emirates.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Arab Emirates. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tolerability to ECP procedures (time frame: Weeks 0-24). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06703333, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2026.