Extracorporeal Photopheresis to Treat Chronic Graft-Versus-Host Disease

Phase 2

Completed

• Conditions: Graft vs Host Disease

• Registration Number: NCT00048789
• Lead Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)

Brief Summary

This study will examine the safety and effectiveness of extracorporeal photopheresis (ECP) for treating chronic graft-versus-host disease (GvHD). GvHD is a common complication of stem cell transplantation using donated stem cells. It occurs when the donor's T-lymphocytes (a type of immune cell) see the patient's cells as foreign and mount an immune response to reject them. The attack can cause skin rash, mouth sores, liver or lung inflammation, lack of appetite, and muscle stiffness. Chronic GvHD can cause serious illness, and even death, from the long-term effects of immune dysfunction and from toxic effects of medications (such as cyclosporine and prednisone) used to treat it.

ECP is an experimental treatment designed to stop the lymphocytes from attacking the body. It involves collecting some of the cells that cause GvHD, treating them with a combination of drug and light therapy and returning them to the body. Sixty to 80 percent of patients with chronic GvHD improve with ECP treatment, and some patients can stop treatment with prednisone or cyclosporine, or reduce the drug dosages.

Patients with chronic GvHD whose condition has not improved after a minimum 14-day course of cyclosporine and prednisone may be eligible for this study. Patients must be able to travel to the NIH Clinical Center in Bethesda, Maryland, twice a week during the 3-month study period.

Upon entering the study, participants will have a baseline evaluation to measure the extent of GvHD. This assessment includes blood tests, eye and dental examinations, skin biopsy for patients with skin involvement, and CT scans and lung function tests to look for possible lung involvement. Biopsies of the lung, liver, mouth, or eye may be requested if needed to confirm GvHD in these tissues. The skin will be photographed before starting ECP treatment and once a month during the treatment period. Following baseline tests, participants will undergo treatment and evaluations as follows:

ECP Treatment

Patients will have blood drawn to collect lymphocytes causing GvHD. This may be done with a special needle or catheter (tube inserted into a vein) or for patients who need or prefer it with a temporary central venous catheter similar to that used for the stem cell transplantation. Patients will have three 2- to 3-hour treatments a week for the first week and two treatments a week after that for a total of 25 treatments over 3 months. Patients who do not tolerate the treatment...

Detailed Description

This is a phase 2 study of extracorporeal photopheresis (ECP) for treatment of chronic graft versus host disease following allogeneic hematopoietic stem cell transplantation. Extracorporeal exposure of human mononuclear cells to ultraviolet A radiation following photosensitization with 8-methoxypsoralen has proven to be an effective treatment for cutaneous T-cell lymphoma. Small, single institution studies have suggested efficacy in other T-cell mediated diseases such as solid organ rejection and graft versus host disease. This a phase 2 study of ECP for treatment of chronic graft versus host disease. Eligible patients must have objective evidence of chronic graft versus host disease that is refractory to conventional therapy or must have steroid-dependent disease that does not permit steroid dose reduction. The primary objective of this study is to better define the safety, efficacy and mechanism of action of ECP for treatment of patients with chronic graft versus host disease.

Study Timeline

• Study Start: 2002-11-04
• Study First Submitted: 2002-11-07
• Study First Submitted QC: 2002-11-07
• Study First Posted: 2002-11-08
• Status Verified: 2009-01-29
• Study Completion: 2009-01-29
• Last Update Submitted: 2017-06-30
• Last Update Posted: 2017-07-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Determination of response rate including degree of improvement; steroid sparing effect; types of cGVHD problems which respond to this therapy; assessment of problems or side effects or adverse events associated with the procedure.

Trial Locations

Locations (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike; 🇺🇸 Bethesda, Maryland, United States