Extracorporeal Photopheresis in Early Diffuse Cutaneous Systemic Sclerosis

NCT04986605 | Not Yet Recruiting Phase 2 FDA Drug FDA Device

• 1 other identifier: ECR-CAN-011068
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess feasibility, safety and preliminary efficacy of Extracorporeal Photopheresis in the treatment of active diffuse cutaneous systemic sclerosis (dcSSc). This pilot study will help to determine if further study (a RCT) is justified.

Conditions

Diffuse Cutaneous Systemic Sclerosis

Keywords

systemic sclerosis; scleroderma; extracorporeal photopheresis; connective tissue diseases; skin diseases

Outcome Measures

Primary Outcomes (1)

• Change in skin thickness measured by modified Rodnan Skin Score

  modified Rodnan Skin Score (mRSS): is a standard outcome measure for skin disease in SSc and calculated by measuring skin thickness in 17 different body sites (each site scored 0-3, with a total possible additive score of 51). A higher skin score (or a higher "skin thickness") and progression of this score, is predictive of internal organ involvement and mortality. While a lower or improving (lessening) score is associated with favorable outcomes, including better survival.

  48 weeks

Secondary Outcomes (11)

• Change in the modified Rodnan Skin Score

  12, 24 and 36 weeks

• Combined Response Index in diffuse cutaneous systemic sclerosis score

  24 weeks

• Change in Forced Vital Capacity

  6 and 12 months

• Change in the diffusing capacity for carbon monoxide

  6 and 12 months

• Change in physician global assessment of disease activity

  12, 24, 36 and 48 weeks

Other Outcomes (6)

• Regimen-related toxicities

  assessed for duration of treatment up to 48 weeks, and up to 12 weeks post-treatment

• Infectious complications

  assessed for duration of treatment up to 48 weeks, and up to 1 month post-treatment

• Change in peripheral levels of T-cell activation marker - sIL-2R

  12, 24, 36 and 48 weeks

Study Arms (1)

Administration of Extracorporeal Photopheresis Treatment

EXPERIMENTAL

Duration of treatment: 48 weeks. Treatments occur on 2 consecutive days every 4 weeks. Dose of UVADEX: Treatment Volume x 0.017 = mL of UVADEX for each treatment Treatment Volume (TV) is defined as: The total volume of Buffy Coat plus prime solution that will undergo photoactivation. Route of administration: Extracorporeal

Device: Extracorporeal Photopheresis (ECP); Drug: UVADEX

Interventions

Extracorporeal Photopheresis (ECP) DEVICE

Drug Intervention using a medical device. The ECP device is already licensed in Canada. License No.7703. ECP treatment, using the drug UVADEX, will be given on 2 consecutive days every 4 weeks for a total of 26 treatment days (48 weeks).

Also known as: ECP, 8-mop

Administration of Extracorporeal Photopheresis Treatment

UVADEX DRUG

The phase II aspect of the study refers to the drug, methoxsalen. Methoxsalen is being used off label from the currently approved indications in the monograph. The study is proposing to use methoxsalen in combination with with extracorporeal photopheresis for the treatment of diffuse cutaneous systemic sclerosis. Treatment will be given in addition to standard of care medications for SSc.

Also known as: 8-mop

Administration of Extracorporeal Photopheresis Treatment

Eligibility Criteria

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

You may qualify if:

• Patients with SSc, aged 18 years or older, and:
• Subjects must meet the ACR/EULAR classification criteria for SSc (2013).
• Early dcSSc (within 5 years of first non-Raynaud's phenomenon symptom) or any other dcSSc patients who have at least one of the signs of disease activity: mRSS of 15 or more, presence of tendon friction rubs, elevated inflammatory markers thought to be due to active dcSSc and not related to other issues such as infection or ILD with FVC% predicted \<80% or HRCT showing ILD thought to be from SSc.
• Able to give informed consent.

You may not qualify if:

• Poor pulmonary function (FVC\<40% and/or DLCO\<30%).
• Class IV PAH or PH.
• Clinically significant cardiac disease.
• Significant concurrent, uncontrolled medical condition including, but not limited to, renal, cardiac, hepatic, pancreatic, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease; and cancer (i.e. co-existing melanoma, basal cell, or squamous cell skin carcinoma).
• Chronic or ongoing active infectious disease requiring systemic treatment, including active tuberculosis (TB) infection.
• Seropositivity for human immunodeficiency virus (HIV) at study entry.
• Active viral infection with viral replication of hepatitis B or C virus at study entry.
• Thrombophilia.
• Contraindications to heparin including history of heparin-induced thrombocytopenia (HIT) or heparin-induced thrombocytopenia and thrombosis (HITTS), history of thrombocytopenia with pentosan polysulfate, known hypersensitivity to heparin or pork products.
• Low Platelet count (less than 100,000 per mm3).
• Aphakia (absence or loss of the eye's lens and has not been replaced with an artificial lens), because of the significantly increased risk of retinal damage due to the absence of lenses.
• Severe anemia (hemoglobin \<70g/L).
• High white blood cell count (greater than 25000 mm3).
• A history of surgical spleen removal.
• A history of a light sensitive disease state, i.e. lupus erythematosus, porphyria cutanea tarda, erythropoietic protoporphyria, variegate porphyria, xeroderma pigmentosum and albinism.

Sponsors & Collaborators

• London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's: lead
• Mallinckrodt: collaborator

Study Sites (1)

Rheumatology Clinic, St. Joseph's Health Care

London, Ontario, N6A4V2, Canada

Location

Related Publications (29)

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MeSH Terms

Conditions

Scleroderma, Diffuse; Scleroderma, Systemic; Connective Tissue Diseases; Skin Diseases

Interventions

Photopheresis; Methoxsalen

Condition Hierarchy (Ancestors)

Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

PUVA Therapy; Ultraviolet Therapy; Phototherapy; Therapeutics; Extracorporeal Circulation; Surgical Procedures, Operative; Furocoumarins; Coumarins; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds, 3-Ring

Study Officials

• Dr. Janet E Pope, MD PhD

  University of Western Ontario, Division of Rheumatology, St. Joseph's Health Care, London, Ontario, Canada

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dr. Janet E Pope, MD PhD

CONTACT

519-646-6332; janet.pope@sjhc.london.on.ca

Amanda Philip

CONTACT

519-646-6000; Amanda.Philip@sjhc.london.on.ca

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Rheumatology

Study Record Dates

• First Submitted: June 1, 2021
• First Posted: August 3, 2021
• Study Start: July 1, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2027
• Last Updated: March 13, 2025

Record last verified: 2025-03

Locations

CA (1)