Interim Data for Phase 3 Graft-versus-Host Disease Trial Looks Promising

According to a story from PR Newswire, the specialty pharmaceutical company Mallinckrodt plc recently announced the release of interim data from the company’s Phase 3 clinical trial. This trial is testing the company’s Therakos Photopheresis platform alongside liquid methoxalen as a treatment for acute graft-versus-host disease (GvHD) in pediatric patients. The results of this trial are mostly encouraging.

About Graft-versus-Host Disease

Graft-versus-Host disease is a potentially fatal medical complication that can appear in a patient that has recently received a transplant operation from another person. It is most commonly triggered by bone marrow transplants but it can also occur with solid organ transplants as well. The disease occurs when white blood cells that remain in the transplanted tissue begin to identify the host body as a foreign intruder and begin to attack the host’s cells. Many recipients of transplanted tissue have weakened immune systems which means that their own body is often incapable of preventing the attack from beginning. Symptoms of graft-versus-host disease include skins rashes, gastrointestinal and liver damage, and damage to the mucosa. Long term disease may see the attack spread to other areas as well, such as the host immune system, the exocrine glands, lungs, and connective tissue. Treatment often includes immunosuppressants such as steroids and certain chemotherapy agents. To learn more about graft-versus-host disease, click here.

Clinical Trial Findings

The clinical trial was tested on pediatric patients whose disease had already stopped responding to steroid therapy. In this study, an analysis of the patients four week 74 percent. This far exceeded the minimum target set for this period of 48 percent. Treatment protocol for this Phase 3 trial allows the company to halt the trial if the response rate is 48 percent or greater, so Mallinckrodt will no longer be enrolling new patients for it. 

Unfortunately, the study saw a total of eleven severe adverse events occur during the course of this trial. These events were not linked to the use of the Therakos Photopheresis immunotherapy platform. These events resulted in two patients withdrawing from the trial and two patient deaths.

To learn more about the results of this study, click here.


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