UVADEX Sterile Solution in Conjunction With the UVAR XTS Photopheresis System as an Interventional Therapy for the Treatment Of CTCL (Mycosis Fungoides) in Patients With TMN Classification Stage 1A, 1B, 2A
Objectives: The study objective is to demonstrate that the UVADEX® Sterile Solution
formulation of methoxsalen used in conjunction with the UVAR XTS Photopheresis System can
have a clinical effect on the skin manifestations of CTCL (mycosis fungoides) in early stage
Methodology: Single-arm, open-label treatment using UVAR XTS Photopheresis System. Treatment
consists of two photopheresis treatments on successive days every 4 weeks for six months.
Those patients completing the first 6-month period may be continued on photopheresis for a
6-month follow-up period. Patients who do not respond to photopheresis therapy after 6
months may have concurrent therapy with low dose bexarotene and interferon added as outlined
in the protocol.
Number of Patients (Planned and Analyzed): The study plan is for a minimum of 50 patients
Diagnosis and Main Criteria for Inclusion: Male or female patients with CTCL diagnosis of
stage IA, IB or IIA with measurable skin lesions (patches or plaques) and a minor blood
abnormality. Patients must be refractory to at least one treatment for early stage CTCL
such as PUVA, Electron beam, oral steroids, high potency topical steroid, topical nitrogen
mustard, methotrexate, interferon, or bexarotene.
Test Product, Dose and Mode of Administration, Batch or Lot Number: UVADEX liquid
methoxsalen 20mcg/mL in conjunction with the UVAR XTS Photopheresis System. UVADEX is
injected into the photoactivation bag during photopheresis therapy in accordance with the
approved drug package insert and UVAR XTS operator's manual. UVADEX dose is less than 200mcg
Duration of Treatment: The study will consist of 2 treatment periods, a 6-month initial
period and a 6-month follow-up period where photopheresis therapy may continue.
Criteria for Evaluation:
Efficacy: The primary endpoint will be the overall response based on skin-weighted
assessment. Secondary endpoints will also include time to response, duration of response,
and a "Quality of Life " assessment. The size and number of lymph nodes and flow cytometry
analyses will also be considered. Experienced skin observers will perform skin scores on
each patient at enrollment. Skin scores will be recorded as the percentage of the patient's
body involved with patch or plaque lesions. A successful response to therapy will be
patients who have a greater than 50% improvement in skin involvement (PR) or complete skin
improvement (CR) without worsening in nodes, blood or visceral organs. Patients with
25%-50% improvement will be considered as minor response (MR), + or - 25% will be SD, and PD
will be defined as 25% worsening from baseline.
Safety: Safety is assessed by the incidence and intensity of adverse events, whether
clinical or based on laboratory results.
Statistical Methods: The primary endpoint will be the proportion of patients who have CR and
PR of their skin lesions.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
The primary endpoint will be the overall response based on skin-weighted assessment.
The overall skin repsonse will be assessed at 6month and will consist of an experts assessment of the % of skin involvement at baseline and 6 months.
Madeleine Duvic, MD
M.D. Anderson Cancer Center
United States: Institutional Review Board
|MD Anderson Cancer Center||Houston, Texas 77030-4096|
|Boston Medical Center||Boston, Massachusetts 02118|
|Vanderbilt University Medical Center||Nashville, Tennessee 37232-2516|
|University of Minnesota||Minneapolis, Minnesota 55455|
|University of Pittsburgh||Pittsburgh, Pennsylvania 15261|
|Rush-Presbyterian Hospital||Chicago, Illinois 60612|
|University Hospital of Cleveland/Case Western Reserve University||Cleveland, Ohio 44106|