Safety and Efficacy of Extracorporeal Photopheresis (ECP) in the Treatment of Multiple Sclerosis

NCT05168384 | Active Not Recruiting Phase 1 DMC

• 2 other identifiers: CT.005.2.0.PHOMSDOH/CVDC/2022/155
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

PHOMS Study is a randomized, controlled, open-label, prospective, and multicentric clinical trial involving outpatients diagnosed with Secondary Progressive Multiple Sclerosis (SPMS) or Relapsing-Remitting Multiple Sclerosis (RRMS). The primary objective is the safety profile assessment of the investigational intervention (Extracorporeal Photopheresis -ECP) and its preliminary efficacy evaluation, while the secondary objective is the assessment of the immune response profile in MS patients.

Conditions

Multiple Sclerosis, Secondary Progressive; Multiple Sclerosis; Multiple Sclerosis, Relapsing-Remitting

Keywords

Extracorporeal Photopheresis; Multiple Sclerosis; Relapsing-Remitting Multiple Sclerosis; Secondary Progressive Multiple Sclerosis

Outcome Measures

Primary Outcomes (9)

• Tolerability to ECP procedures (Group A patients)

  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)

  Proportion of patients referring TEAEs, AESIs, and SAEs assessed by CTCAE v5.0.

  Weeks 0-52

• Tolerability to TEAEs, AESIs, and SAEs

  Proportion of patients tolerating TEAEs, AESIs, and SAEs, and finalizing the Study

  Weeks 0-52

• Clinical improvement (25-foot walk)

  Proportion of patients with clinical improvement from baseline in 20% or greater increase in the timed 25-foot walk

  Baseline, months 3, 6, 9, and 12

• Clinical improvement (9-hole peg test)

  Proportion of patients with clinical improvement from baseline in 20% or greater increase in the 9-hole peg test

  Baseline, months 3, 6, 9, and 12

• Clinical improvement (36-Item Short Form Survey)

  Proportion of patients with clinical improvement from baseline in 20% or greater increase in the 36-Item Short Form Survey (SF-36)

  Baseline, months 3, 6, 9, and 12

• Clinical improvement (EDSS baseline low score)

  Proportion of patients with clinical improvement from baseline in 1 point or greater increase in EDSS score (in subjects with baseline EDSS scores between 3 and 5.5)

  Baseline, months 3, 6, 9, and 12

• Clinical improvement (EDSS baseline high score)

  Proportion of patients with clinical improvement from baseline in 0.5 point or greater increase in EDSS score (in subjects with baseline EDSS scores ≥ than 6)

  Baseline, months 3, 6, 9, and 12

• Occurrence of clinical relapse at any point in the study

  Proportion of patients demonstrating new or recurrent neurological symptoms consistent with MS, symptoms last 24 to 48 hours, or development of new MS symptoms over days to weeks

  Weeks 0-52

Secondary Outcomes (2)

• Immune response profile (cellular)

  Baseline, months 3, 6, 9, and 12

• Immune response profile (humoral)

  Baseline, months 3, 6, 9, and 12

Study Arms (2)

Group A (ECP + SoC Group)

EXPERIMENTAL

Extracorporeal photopheresis (ECP) plus Multiple Sclerosis (MS) standard of care

Combination Product: Extracorporeal Photopheresis; Combination Product: MS standard of care

Group B (SoC Group)

ACTIVE COMPARATOR

MS standard of care alone (SoC, defined by Disease-modifying Therapy -DMT, recommended by the American Academy of Neurology -AAN

Combination Product: MS standard of care

Interventions

Extracorporeal Photopheresis COMBINATION_PRODUCT

ECP procedures will be performed using a Therakos Cellex integrated, closed photopheresis system (Therakos, Inc., a Mallinckrodt Pharmaceuticals Company). ECP will administered according to the following schedule (Group A): Weeks 1-8: Twice per week (16 sessions). Weeks 9-16: Once per week (8 sessions). Weeks 17-24: Once every 2 weeks (4 sessions). Total: 28 sessions (within 24 weeks).

Also known as: Extracorporeal Photoimmunotherapy, Photochemotherapy

Group A (ECP + SoC Group)

MS standard of care COMBINATION_PRODUCT

Disease-modifying Therapy -DMT, recommended by the American Academy of Neurology -AAN

Also known as: Disease-modifying Therapies

Group A (ECP + SoC Group); Group B (SoC Group)

Eligibility Criteria

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

You may qualify if:

• Demonstrate Expanded Disability Status Scale (EDSS) scores between 3 to 6.5 at screening.
• Documented EDSS progression in the 2 years prior to screening of 1 point or greater for patients with an EDSS score less than 6 at baseline, and greater than or equal to 0.5 for patients with an EDSS score greater than or equal to 6.0 at baseline \*.
• \* If documented EDSS scores are not available, a written summary of the clinical evidence of disability progression over the last 2 years, and retrospective assessment of EDSS score from data in the medical records, must be submitted for review by the principal investigators.
• Documented initial onset characterized by a relapsing-remitting course as described in the Diagnostic Criteria.
• Age ≥ 18 ≤ 75 years.
• Weight \> 40 kg.
• Hematocrit ≥ 28 % (with or without transfusion support).
• Platelet count \> 100,000 per μL (with or without transfusion support).
• Willingness to use at least 1 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 PHOMS Study tests, visits, and procedures (including the ECP), as outlined in the informed consent.
• Patients must have adequate peripheral venous access to initiate ECP therapy, and central line insertion shall be required.
• The patient agrees to participate in the trial and signs the PHOMS Study informed consent form.

You may not qualify if:

• Absolute medical contraindication to receive ECP.
• Laboratory evidence of any of the following:
• White blood cells (WBC) \< 2,000 cells per uL.
• Serum transaminase levels \> x 2 UNL.
• Creatinine Clearance \< 60 mL/min.
• Concurrent diagnosis of a neurological condition that would interfere with the assessment of MS, or an autoimmune disease or inflammatory condition that is chronically treated with immunosuppressive agents.
• Evidence of known infection with human immunodeficiency virus (HIV) or active (not including latent) Hepatitis B.
• Uncontrolled infection requiring treatment at study entry.
• Hypersensitivity or allergy to psoralen (methoxalen).
• Inability to tolerate fluid changes associated with ECP (e.g., inadequate renal, hepatic, pulmonary and cardiac function leading to enable patient to tolerate extracorporeal volume shifts associated with ECP).
• Presence of aphakia or photosensitive disease (systemic lupus erythematosus, porphyrias, etc.).
• Women who are pregnant and/or lactating.
• 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 pharmodynamic effect has returned to baseline, whichever is longer.
• Inability to undergo MRI scans.
• Contraindication to gadolinium due to past allergic, hypersensitive, or adverse reaction or impaired renal function. Patients receiving a steroid prep prior to gadolinium administration due to history of hypersensitivity or allergy to other agents or due to prior mild reaction to gadolinium will not be excluded from the study.

Sponsors & Collaborators

• Abu Dhabi Stem Cells Center: lead

Study Sites (1)

Abu Dhabi Stem Cells Center

Abu Dhabi, Abu Dhabi Emirate, 4600, United Arab Emirates

Location

Related Publications (2)

• Castillo-Aleman YM, Villegas-Valverde CA, Ventura-Carmenate Y, Al-Kaabi FM, Lumame S, Castelo C, Mir R, Brylev L, Atieh M, Haider MT, Bencomo-Hernandez AA. Mononuclear cell recruitment during extracorporeal photopheresis: Partial results of a phase 1/2 randomized clinical trial in multiple sclerosis. Transfus Apher Sci. 2025 Apr;64(2):104095. doi: 10.1016/j.transci.2025.104095. Epub 2025 Feb 9.

  PMID: 39947046 BACKGROUND

• Castillo-Aleman YM, Lumame S, Castelo C, Mir R, Ventura-Carmenate Y, Al-Kaabi FM. Recurrent clumping in the extracorporeal photopheresis circuit using acid citrate dextrose solution A. J Clin Apher. 2024 Jun;39(3):e22117. doi: 10.1002/jca.22117. No abstract available.

  PMID: 38661254 BACKGROUND

MeSH Terms

Conditions

Multiple Sclerosis; Multiple Sclerosis, Relapsing-Remitting; Multiple Sclerosis, Chronic Progressive

Interventions

Photopheresis; Photochemotherapy

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

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

Study Officials

• Yendry Ventura Carmenate, M.D.

  Abu Dhabi Stem Cells Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 25, 2021
• First Posted: December 23, 2021
• Study Start: March 26, 2022
• Primary Completion: September 1, 2025
• Study Completion: May 1, 2026
• Last Updated: April 9, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AE (1)