Extracorporeal Photopheresis for Systemic Sclerosis

Overseen By Amanda Philip

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: London Health Sciences Centre Research Institute and Lawson Research Institute of St. Joseph's

Must not be taking: Photosensitizing drugs, Immunosuppressants

Disqualifiers: Cardiac disease, Cancer, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 48 weeks

15 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a treatment called Extracorporeal Photopheresis for patients with a severe form of scleroderma. The treatment involves taking blood out, treating it with light and a drug, and then putting it back in. The goal is to see if this can safely reduce skin thickening and improve symptoms. Extracorporeal photopheresis (ECP) has been used for over 30 years, initially for cutaneous T-cell lymphoma and later for other diseases including scleroderma.

Do I need to stop my current medications for this trial?

The trial does not specify if you need to stop taking your current medications, but it does mention that patients using more than two immunosuppressants cannot participate. If you are on photosensitizing drugs, you may need to stop those as they are listed in the exclusion criteria.

What data supports the effectiveness of the treatment Extracorporeal Photopheresis (ECP) for Systemic Sclerosis?

Extracorporeal Photopheresis (ECP) has shown clinical benefits in treating systemic sclerosis and similar fibrosing diseases, and it is effective for other conditions like graft versus host disease and autoimmune diseases. Although the exact way it works is not fully understood, studies suggest it can help manage severe cases of systemic sclerosis. ^{1 2 3 4 5}

Is extracorporeal photopheresis (ECP) safe for humans?

Extracorporeal photopheresis (ECP) has been used safely for various conditions, including cutaneous T-cell lymphoma and systemic sclerosis, with studies focusing on its safety and feasibility. While bacterial contamination is a potential risk due to the immunosuppressive nature of the treatment, specific incidence data is not provided. ^{3 4 6 7 8}

How is the treatment Extracorporeal Photopheresis (ECP) unique for systemic sclerosis?

Extracorporeal Photopheresis (ECP) is unique for systemic sclerosis because it involves a process where blood cells are treated with a light-sensitive drug and ultraviolet light before being returned to the body, aiming to modulate the immune system. This approach is different from standard treatments as it targets immune cells directly and has shown benefits in other autoimmune conditions, although its exact mechanism in systemic sclerosis is not fully understood. ^{1 2 3 7 9}

Research Team

Dr. Janet E Pope, MD PhD

Principal Investigator

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

Eligibility Criteria

This trial is for adults with early diffuse cutaneous systemic sclerosis (dcSSc) within 5 years of their first symptoms, or those showing active disease signs. Participants must not be in other trials recently, have severe anemia or low platelets, HIV, certain heart/lung conditions, uncontrolled diseases like cancer or infections, and must not be pregnant or using certain drugs.

Inclusion Criteria

I have early or active diffuse cutaneous systemic sclerosis.

I am 18 or older with systemic sclerosis.

You must meet the specific classification criteria for systemic sclerosis set by ACR/EULAR in 2013.

Exclusion Criteria

I do not have an ongoing infection needing treatment.

I have a blood clotting disorder.

I have a serious heart condition.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Extracorporeal Photopheresis treatment for diffuse cutaneous systemic sclerosis

48 weeks

Treatments occur on 2 consecutive days every 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Treatment Details

Interventions

• Extracorporeal Photopheresis (ECP)
• Extracorporeal Photopheresis (ECP) with Methoxsalen
• UVADEX

Trial Overview The study tests the safety and initial effectiveness of Extracorporeal Photopheresis (ECP) using UVADEX for treating dcSSc. It's a pilot study to see if more extensive research is needed. ECP involves treating blood with light after adding a drug that makes cells sensitive to light.

Participant Groups

Group I: Administration of Extracorporeal Photopheresis Treatment Experimental Treatment 2 Interventions

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

Who Is Running the Clinical Trial?

London Health Sciences Centre Research Institute and Lawson Research Institute of St. Joseph's

Lead Sponsor

Trials

686

Recruited

427,000+

London Health Sciences Centre OR Lawson Research Institute of St. Joseph's

Lead Sponsor

Trials

668

Recruited

424,000+

London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

Lead Sponsor

Trials

678

Recruited

421,000+

Lawson Health Research Institute

Lead Sponsor

Trials

684

Recruited

432,000+

Mallinckrodt

Industry Sponsor

Trials

202

Recruited

15,700+

Mario Saltarelli

Mallinckrodt

Chief Medical Officer

MD

Siggi Olafsson

Mallinckrodt

Chief Executive Officer

PhD in Pharmacy

Findings from Research

In a study involving 14 patients with severe atopic dermatitis, long-term extracorporeal photochemotherapy (ECP) resulted in complete clinical remission for 29% of participants and substantial improvement for 36%, indicating its efficacy as a treatment option.

The treatment was found to be safe, with no significant adverse effects or signs of immunosuppression reported, suggesting that ECP could be a viable option for patients with severe and resistant cases of atopic dermatitis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term application of extracorporeal photochemotherapy in severe atopic dermatitis. Prinz, B., Michelsen, S., Pfeiffer, C., et al.[2019]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Management of severe scleroderma with long-term extracorporeal photopheresis. [2018]

2. United States pubmed.ncbi.nlm.nih.gov

Photopheresis: Advances and Use in Systemic Sclerosis. [2018]

3. United States pubmed.ncbi.nlm.nih.gov

Extracorporeal photochemotherapy does not suppress T- or B-cell responses to novel or recall antigens. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Long-term application of extracorporeal photochemotherapy in severe atopic dermatitis. [2019]

5. Switzerland pubmed.ncbi.nlm.nih.gov

[Extracorporeal photopheresis]. [2019]

6. Italy pubmed.ncbi.nlm.nih.gov

Feasibility and safety of a new technique of extracorporeal photochemotherapy: experience of 240 procedures. [2007]

7. United States pubmed.ncbi.nlm.nih.gov

Extracorporeal photopheresis: clinical use so far. [2012]

8. United States pubmed.ncbi.nlm.nih.gov

Bacterial contamination rates in extracorporeal photopheresis. [2021]

9. England pubmed.ncbi.nlm.nih.gov

Preliminary results on long-term follow-up of systemic sclerosis patients under extracorporeal photopheresis. [2022]