Photopheresis in Early-stage Mycosis Fungoides

Official title

THERAKOS® CELLEX Photopheresis System as an Interventional Therapy for the Treatment of Early Stage CTCL (Mycosis Fungoides), an Open-label, Single-arm, Multi-center, Phase II Study

Overview

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 (CTCL). Currently, photopheresis is performed as a palliative treatment for late stage CTCL. However, recent studies have demonstrated that patients with early stage CTCL may have markers in their blood which were previously observed primarily in late stage disease, such as clonal T cell populations. Considering these findings, the study aims to investigate whether photopheresis therapy may be used earlier in the disease course to produce a clinical response.

Detailed description

The THERAKOS® CELLEX Photopheresis System is currently FDA approved and indicated for the palliative treatment of the skin manifestations of cutaneous T-cell lymphoma (CTCL) in patients not responsive to other forms of treatment. The original studies in CTCL were performed in patients having extensive patch/plaque or erythrodermic stage disease. Therefore, photopheresis is used primarily to treat stage III or IV CTCL. However, studies have shown that 50% of patients with early disease may have clonal T-cells in their peripheral blood if polymerase chain reaction is used to amplify the T-cell receptor gamma gene fragments. Thus, patients who are staged as T1 or T2 who also have slight abnormalities in blood involvement, such as atypical cells seen on blood smear or abnormal flow cytometry may benefit from photopheresis therapy. This study aims to investigate the efficacy of photopheresis therapy in patients with Stage !A, 1B, or IIA CTCL.

Interventions

• Drug UVADEX® (methoxsalen) Sterile Solution in conjunction with the THERAKOS® CELLEX Photopheresis
  Extracorporeal Photopheresis (ECP)
• Device THERAKOS® CELLEX photopheresis system
  THERAKOS® CELLEX is an FDA-approved extra-corporeal photopheresis system

Primary outcome measures

• Overall Response Rate [Time frame: 1 Year]

Secondary outcome measures (7)

• Time to Response of Photopheresis Therapy [Time frame: 1 Year]
• Duration of Response [Time frame: 1 Year]
• Change in Functional Assessment of Cancer Therapy - General (FACT-G) Score [Time frame: Baseline and 1 Year]
• Change in Skindex-29 Score [Time frame: Baseline and 1 Year]
• Change in Short Form Survey (SF-36v2) Score [Time frame: Baseline and 1 Year]
• Change in Size of Lymph Nodes [Time frame: Baseline and 1 Year]
• Change in Sézary Cell Counts at 6 and at 12 months [Time frame: Baseline, 6 months and 12 months]

Eligibility criteria

Inclusion criteria

• Who are male or female, over the age of 18 and <40 kg body weight with adequate veins to provide intravenous access.
• Who are willing to adhere to the protocol and sign an Informed Patient Consent Document
• Must not be on any other investigational device/drug treatment.
• Who have the diagnosis of Mycosis Fungoides (MF) including a skin biopsy consistent with MF (atypical epidermotropic or folliculocentric T-cells) with appropriate staging as IA, IB or IIA: T1 or T2 (patches or plaques) with measurable lesions.
• With IA stage must demonstrate a minor blood abnormality by morphology/laboratory assessment.
• With IIA stage - clinically significant nodes (1.5 cm) must have lymph node biopsy showing dermatopathic nodes or no involvement.
• Must be willing and able to discontinue concomitant medications for MF. Subjects currently taking the following drugs must discontinue medication with the following wash out periods prior to enrollment in the trial: PUVA or UVB Therapy - 4 weeks, topical nitrogen mustard or other topical chemotherapy - 4 weeks, bexarotene capsules or other systemic biologic agent - 3 weeks washout, high dose topical steroids, topical retinoids or immunotherapy - 2 week washout with 1% topical hydrocortisone , oral steroids above 10 mg - 30 day washout, unless subject has Addison's Disease or adrenal insufficiency
• Who are refractory to at least one of the standard therapies used to treat Stage IA, IB or IIA CTCL such as oral steroids, high-dose topical steroids, topical nitrogen mustard, Bexarotene, PUVA therapy, electron beam radiation, biological response modifiers or oral methotrexate.
• Must be willing to abstain from therapeutic sunbathing, phototherapy, tanning beds, etc. for the duration of the study.

Exclusion criteria

• Who have MF (T3 cutaneous tumors or T4 exfoliative erythroderma) Stage IIB - IVB
• Who are unable to tolerate extracorporeal volume loss i.e., severe cardiac disease/anemia
• With deterioration of renal function who have a serum creatinine level greater than 3.0 mg/dL.
• With lipemic plasma >500 ng/dL, uncontrolled diabetes, history of liver damage (2.5 x normal alanine transaminase (ALT), aspartate aminotransferase (AST), porphyria, lupus, positive tests for human immunodeficiency virus (HIV) antibody, hepatitis C virus (HCV) antibody or Hepatitis B Surface Antigen, severe emotional, behavioral or psychiatric problems that, in the opinion of the investigator, would result in poor compliance with the treatment regimen, and idiosyncratic or hypersensitivity reactions to 8-methoxypsoralen compounds, heparin, or citrate.
• On oral prednisone therapy or high potency topical steroids.
• Who are pregnant or nursing a child.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

United States · 1 center

• Columbia University Irving Medical Center — New York

Identifiers

NCT: NCT05680558 · AAAS9960

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗