Photopheresis for Mycosis Fungoides

Overseen By Larisa J Geskin, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Columbia University

Must not be taking: Oral prednisone, High potency steroids

Disqualifiers: Severe cardiac disease, Uncontrolled diabetes, 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: Unknown

Duration: 12 months

74 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether photopheresis therapy can benefit individuals with early-stage cutaneous T-cell lymphoma (CTCL), a type of skin cancer. While typically used for advanced cases, researchers aim to determine if earlier intervention can improve outcomes. The treatment uses the THERAKOS® CELLEX photopheresis system and a special light-sensitive solution called UVADEX® (methoxsalen). Suitable candidates have early-stage CTCL with measurable skin patches or plaques and have not responded to other treatments. Participants must stop certain medications and avoid activities like sunbathing during the study. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

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

Yes, you will need to stop taking certain medications for Mycosis Fungoides before joining the trial. There are specific 'washout' periods (time without taking certain medications) required: 4 weeks for PUVA or UVB therapy and topical chemotherapy, 3 weeks for bexarotene capsules, 2 weeks for high dose topical steroids, and 30 days for oral steroids above 10 mg, unless you have Addison's Disease or adrenal insufficiency.

What prior data suggests that the THERAKOS® CELLEX photopheresis system is safe for treating early stage cutaneous T-cell lymphoma?

Studies have shown that the THERAKOS CELLEX photopheresis system has FDA approval for treating skin problems in advanced cutaneous T-cell lymphoma (CTCL), confirming its safety. Patients generally tolerate the treatment well. It employs extracorporeal photopheresis (ECP), which strengthens the immune system.

Research indicates that UVADEX (methoxsalen) Sterile Solution, used with photopheresis, also maintains a good safety record. It is generally well-tolerated and has been used in similar treatments. However, side effects like sensitivity to sunlight or skin reactions can occur, and only doctors experienced in treating CTCL should use it.

Overall, both treatments have undergone testing and are considered safe for specific conditions. Prospective clinical trial participants should discuss potential risks and benefits with their doctor to make an informed decision. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for mycosis fungoides?

Unlike the standard treatments for mycosis fungoides, which often involve topical steroids, chemotherapy, or radiation, the THERAKOS® CELLEX Photopheresis System in combination with UVADEX® offers a unique approach. This system uses photopheresis, a process that collects and treats a patient's blood cells with a special light-sensitizing agent, methoxsalen, followed by UVA light exposure. This method targets the immune system to fight cancer cells more precisely, potentially offering fewer side effects. Researchers are excited because this treatment may enhance the body's natural ability to combat the disease with a novel mechanism that differs from conventional therapies.

What evidence suggests that photopheresis might be an effective treatment for early stage cutaneous T-cell lymphoma?

Research shows that photopheresis, using the THERAKOS® CELLEX system with UVADEX® (methoxsalen), can effectively treat cutaneous T-cell lymphoma (CTCL), a type of skin cancer. In past studies, many patients experienced significant improvements in their skin condition with this treatment. Real-world data indicates that photopheresis often succeeds, with some reports showing a 77% overall response rate in CTCL patients. Additionally, some patients have maintained improvements for up to five years. This treatment works by engaging the body's immune system to improve skin symptoms in early-stage CTCL. Participants in this trial will receive the UVA Sterile Solution alongside the THERAKOS® CELLEX Photopheresis System. ^{1 3 6 7 8}

Who Is on the Research Team?

Larisa J Geskin, MD

Principal Investigator

Columbia University

Are You a Good Fit for This Trial?

Adults over 18, under 40 kg with early-stage cutaneous T-cell lymphoma (CTCL) and minor blood abnormalities. Participants must not be on other investigational treatments, willing to stop certain medications before joining, and able to give informed consent. Excluded are those on high-dose steroids or with severe health issues like kidney failure, uncontrolled diabetes, liver damage, or infectious diseases like HIV.

Inclusion Criteria

I am willing to stop my current medications for MF with the required washout periods before joining the trial.

Willing to adhere to the protocol and sign an Informed Patient Consent Document

I am not currently participating in any other clinical trials.

Exclusion Criteria

My kidney function is poor with a creatinine level over 3.0 mg/dL.

I am currently taking oral prednisone or using strong topical steroids.

I do not have severe liver issues, uncontrolled diabetes, lupus, porphyria, HIV, hepatitis B or C, severe mental health issues, or allergies to specific medications.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive photopheresis treatment with the THERAKOS® CELLEX Photopheresis System on two consecutive days every 2 weeks for the first 3 months; then once per month for the following 9 months

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• THERAKOS® CELLEX photopheresis system
• UVADEX® (methoxsalen) Sterile Solution

Trial Overview The trial is testing the effectiveness of photopheresis therapy using the THERAKOS® CELLEX system and UVADEX® for improving early-stage CTCL. Photopheresis is usually a palliative treatment for late-stage CTCL but may benefit earlier stages by targeting abnormal blood markers.

How Is the Trial Designed?

Group I: UVA Sterile Solution in conjunction with the THERAKOS® CELLEX Photopheresis System Experimental Treatment 2 Interventions

TREATMENT with THERAKOS® CELLEX Photopheresis System on two consecutive days every 2 weeks for the first 3 months; then once per month for following 9 months.

THERAKOS® CELLEX photopheresis system is already approved in United States, European Union for the following indications:

Approved in United States as THERAKOS CELLEX photopheresis system for:

• Cutaneous T-cell lymphoma (CTCL)
• Sézary syndrome
• Mycosis fungoides

Approved in European Union as Extracorporeal photopheresis for:

• Cutaneous T-cell lymphoma (CTCL)
• Sézary syndrome
• Mycosis fungoides

Who Is Running the Clinical Trial?

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+

Mallinckrodt, Inc.

Collaborator

Trials

1

Recruited

70+

Published Research Related to This Trial

Extracorporeal photo-chemotherapy (ECP) showed an overall response rate of 42% in 50 patients with mycosis fungoides, with a median time to response of 11 months, indicating its efficacy as a treatment option.

ECP was associated with a low incidence of adverse events (16%) and significantly improved overall survival when combined with other therapies, suggesting it is a safe and effective treatment for mycosis fungoides.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Extracorporeal photochemotherapy in mycosis fungoides. Atilla, E., Atilla, PA., Bozdag, SC., et al.[2018]

The THERAKOS CELLEX system for extracorporeal photopheresis (ECP) in patients with cutaneous T-cell lymphoma (CTCL) demonstrated a strong safety profile, with no adverse device effects reported during 155 treatments across 13 patients.

This new system significantly reduced treatment times and extracorporeal volumes, averaging 74.4 minutes for dual-needle treatments compared to 103.0 minutes for single-needle, indicating improved efficiency without compromising safety.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of a new, single, integrated, closed photopheresis system in patients with cutaneous T-cell lymphoma. Bisaccia, E., Vonderheid, EC., Geskin, L.[2013]

In a study of 65 patients with stage III/IV mycosis fungoides, only 35% of African-American patients received extracorporeal photopheresis (ECP) compared to 66% of Caucasian patients, indicating a significant disparity in treatment rates.

The study found that while ECP was discussed as a treatment option with 45% of African-American patients versus 82% of Caucasian patients, once it was discussed, both groups had similar rates of acceptance for the treatment (78% vs 81%).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Racial differences in the use of extracorporeal photopheresis for mycosis fungoides. Agi, C., Kuhn, D., Chung, J., et al.[2018]

Extracorporeal photopheresis (ECP) using the Therakos™ Cellex(®) device is a safe and tolerable second-line treatment for graft-versus-host disease (GVHD) in children and young adults, with a review of 385 procedures showing low rates of complications.

The study found that using prophylactic tissue plasminogen activator reduced central venous line-related occlusions, highlighting a strategy to improve the safety of ECP procedures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The use of novel Therakos™ Cellex® for extracorporeal photopheresis in treatment of graft-versus-host disease in paediatric patients. Rangarajan, HG., Punzalan, RC., Camitta, BM., et al.[2019]

Extracorporeal photopheresis (ECP) combined with other therapies resulted in a 65% overall response rate in 20 patients with erythrodermic mycosis fungoides and Sezary syndrome, with 30% achieving complete response and 35% partial response, lasting over 2 years for 38.5% of patients.

In patients with leukaemic involvement, the blood response to ECP occurred earlier and was maintained longer than the skin response, indicating that a positive blood response does not necessarily predict improvement in skin symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Erythrodermic mycosis fungoides and Sézary syndrome treated with extracorporeal photopheresis as part of a multimodality regimen: A single-centre experience. Atzmony, L., Amitay-Laish, I., Gurion, R., et al.[2016]

The transition to the Therakos® CELLEX® fully integrated system for extracorporeal photopheresis (ECP) significantly increased the number of treatments performed annually from 225 in 2012 to 727 in 2015, demonstrating improved capacity for patient care.

Using the CELLEX® system reduced the treatment time from 270 minutes to 120 minutes per session and lowered the cost per treatment from €1,429.37 to €1,264.70, highlighting both time and cost efficiencies that can enhance patient treatment availability.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A transition from using multi-step procedures to a fully integrated system for performing extracorporeal photopheresis: A comparison of costs and efficiencies. Azar, N., Leblond, V., Ouzegdouh, M., et al.[2021]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05680558

Photopheresis in Early-stage Mycosis Fungoides The purpose of this study is to determine whether photopheresis therapy can be used to improve the clinical course of early stage cutaneous T-cell lymphoma ...

2. therakos.com therakos.com/therakos-photopheresis/prescribing-ecp/

Prescribing ECP for CTCL | THERAKOS® Photopheresis ... Learn about identifying CTCL patients unresponsive to other treatments and prescribing Extracorporeal Photopheresis (ECP) with THERAKOS® Photopheresis.

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38852942/

Chart review study of real-world clinical outcomes in ... This study describes real-world treatment patterns and response rates of extracorporeal photopheresis (ECP) in CTCL patients.

4. tandfonline.com tandfonline.com/doi/full/10.1080/09546634.2024.2360568

Chart review study of real-world clinical outcomes in ... This study describes real-world treatment patterns and response rates of extracorporeal photopheresis (ECP) in CTCL patients.

5. prnewswire.com prnewswire.com/news-releases/new-data-on-patients-with-cutaneous-t-cell-lymphoma-treated-with-extracorporeal-photopheresis-from-a-retrospective-study-presented-at-the-american-society-of-hematology-annual-meeting-301442694.html

New Data on Patients with Cutaneous T-cell Lymphoma ... Therakos immunotherapy is delivered through CELLEX systems to harness the power of the patient's immune system to treat the skin manifestations ...

6. therakos.com therakos.com/therakos-photopheresis/

What is THERAKOS® Photopheresis? The THERAKOS Photopheresis System is an immunomodulatory therapy that uses extracorporeal photopheresis (ECP) to help enhance immunologic response.

7. patient.therakos.com patient.therakos.com/learn-about-ctcl-skin-symptoms/

Learn about CTCL Skin Symptoms CTCL is a type of non-Hodgkin's lymphoma (a type of cancer). It is caused by cells that change in the blood and travel to the skin.

8. therakoshcp.ca therakoshcp.ca/assets/PDF/About%20Therakos%20ECP%20%20Canada%20Presentation%20PRESCRIBER%20(CA-2000094).pdf

Therakos™ ECP EDGE WARNINGS & PRECAUTIONS: THERAKOS Photopheresis treatments should always be performed in locations where standard medical emergency equipment is available.