A Safety and Efficacy Study of Uvadex and Extracorporeal Photopheresis (ECP) in Chronic Graft Versus Host Disease

Condition(s) targeted: Chronic Graft Versus Host Disease

Intervention: ECP(Methoxsalen)+Corticosteroids+Cyclosporine (Drug); Corticosteroids/Cyclosporine (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Therakos

Official(s) and/or principal investigator(s):
Dennis parenti, MD, Study Director, Affiliation: Therakos

Overall contact:
Dennis Parenti, MD, Email: dparent@its.jnj.com

The purpose of this study is to evaluate the safety and effectiveness of extracorporeal photopheresis therapy when added to standard drug therapies administered to patients with moderate to severe chronic graft-versus-host disease.

Official title: A Randomized Controlled Study of Extracorporeal Photopheresis (ECP) Therapy With UVADEX for the Treatment of Patients With Moderate to Severe Chronic Graft-versus-Host Disease (cGvHD)

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Outcomes Assessor), Primary Purpose: Treatment

Primary outcome: Overall response (complete or partial response) in cGvHD according to NIH Response Criteria

Secondary outcome:

Proportion of patients in each treatment group achieving response rates (complete or partial) by organ system involvement

Correlation between total skin score and skin assessment (% of skin involvement)

Quality of Life (QoL) Questionnaire SF 36 short version scores

QoL questionnaire, Bone marrow Transplant scores

Relapse of underlying malignancy

Results from clinical laboratory tests

Adverse Events Reported

Detailed description: This is an open-label study (patients and study staff will know the identity of treatments assigned during the study) in patients with chronic graft-versus-host disease (cGvHD). Chronic graft-versus-host disease (a donator-versus-recipient-disease) is a complication that can occur after a blood stem cell or bone marrow transplant with cells from a related or unrelated donator. During cGvHD, the transplanted cells attack the recipient's body. Patients with cGVHD who meet entry criteria for the study will be randomly assigned to receive standard of care treatment for 26 weeks or standard or care treatment with extracorporeal photopheresis (ECP) for 26 weeks. Standard of care treatment consists of orally (taken by mouth) administered corticosteroids (drugs that reduce inflammation) and cyclosporine (CsA) (a drug that suppresses the patient's immune response). ECP therapy is a process that takes place in a device where the investigational drug UVADEX (methoxsalen) is injected into a germ-free bag mixed with the patient's white blood cells. After the blood cells have absorbed the drug and are exposed to ultraviolet A (UVA) radiation, the blood cells are injected back into the patient's body. During the study, a third party assessor at each study center who will be blinded (will not know) to treatment will assess the condition each patient's skin and oral mucosa at protocol-specified visits and will complete a total skin score for all patients . The study will consist of 3 phases: a screening phase, an open label treatment phase and an end-of-study (or early withdrawal) phase. The duration of patient participation will be 28 weeks and patient safety will be monitored throughout the study.

Minimum age: 18 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Have new onset of moderate or severe cGvHD as assessed by the NIH Consensus Criteria

Clinical Assessment (staging and severity) with onset within 2 years of transplantation (Patients with prior acute GvHD should be on a stable dose of <0. 5 mg/kg daily prednisone, or equivalent, for at least 2 weeks prior to study entry. Prior ECP for patients with acute GvHD is permitted in the study); Be using adequate birth control; If a woman, must have negative pregnancy test result at screening: Be able and willing to comply with all study procedures including providing informed consent

Exclusion Criteria:

- Be intolerant to corticosteroids; Received treatment with 1. 0 mg daily prednisone, or

equivalent, for cGvHD for more than 4 days prior to baseline visit; received treatment with prednisone for mild cGVHD with >30mg/kg for > 30 days, Have evidence of known infection with human immunodeficiency virus (HIV), active Hepatitis B infection, or have uncontrolled infection requiring treatment at the time of study entry; Requires treatment with budesonide and similar low absorption oral steroids and steroid enema preparations; Receiving treatment with mycophenolate mofetil (MMF),PUVA, tyrosine kinase inhibitors, anti-tumor necrosis factor (TNF) agents, sirolimus, and bortezomib; Receiving treatment with alemtuzumab, antithymocyte globulin (ATG) or other similar long-acting agents used for treatment of acute or chronic GvHD or administered during the conditioning regimen <90 days prior to randomization

Dennis Parenti, MD, Email: dparent@its.jnj.com

General Hospital of Vienna (Medical University of Vienna), Vienna, Austria; Recruiting

CHU de Nantes, Nantes, France; Recruiting

Hospital Saint Louis, Paris, France; Recruiting

Universitätsklinik Köln, Cologne 50937, Germany; Recruiting

Universitäts-Klinikum Carl Gustav Carus, Dresden 01309, Germany; Recruiting

Universitatsklinikum Erlangen, Erlangen 91054, Germany; Recruiting

Universitätsklinikum Essen, Essen 45122, Germany; Recruiting

Universitatskrankenhaus Hamburg-Eppendorf, Hamburg 20246, Germany; Recruiting

Universitätsklinikum Leipzig AöR, Leipzig 04103, Germany; Recruiting

Universitätklinikum Mannheim, Mannheim 68167, Germany; Recruiting

Medizinische Universitatsklinik, Munchen 81377, Germany; Recruiting

Uniklinik für Kinder und Jugendmedizin, Tubingen 72076, Germany; Recruiting

Egyesített Szent István és Szent László Kórház, Gyáli út 5-7,, Budapest 1097, Hungary; Not yet recruiting

A.O.U. Policlinico- Vittorio Emanuele, Catania, Italy; Recruiting

Universita de Genova - Ospedale S. Martino, Genova, Italy; Recruiting

Santa Creu I Sant Pau, Barcelona 08025, Spain; Recruiting

Hospital Marques de Valdecilla, Cantabria, Spain; Recruiting

University of Birmingham: Queen Elizabeth Hospital, Birmingham, United Kingdom; Recruiting

Royal Marsden Hospital, London, United Kingdom; Recruiting

University of Miami Hospital, Miami, Florida 33136, United States; Recruiting

Kings College Hospital, London, Greater London SE5 9RS, United Kingdom; Recruiting

Indiana Blood and Marrow Transplantation Research, Indianapolis, Indiana 46237, United States; Recruiting

Tulane University Health Sciences Center, 1430 Tulane Avenue, Rm 7551,, New Orleans, Louisiana 70112, United States; Not yet recruiting

Hackensack University Medical Center, Hackensack, New Jersey 07601, United States; Recruiting

New York Presbyterian Hospital, New York City, New York 10065, United States; Recruiting

Stony brook university medical Center, stony Brook, New York 11794, United States; Recruiting

Hopital Saint Antoine, Saint Antoine, Paris 75571, France; Not yet recruiting

Vanderbilt Ingram Cancer Center, Nashville, Tennessee 37232, United States; Recruiting

Parkland Memorial Hospital, Dallas, Texas 75235, United States; Recruiting

Medical City Dallas Hospital,Transplant center, Dallas, Texas 75230, United States; Recruiting

Methodist healthcare system of san Antonio, San Antonio, Texas 78229, United States; Recruiting

Starting date: November 2011
Last updated: January 14, 2013