A Phase III Study of Remestemcel-L for Children with Acute Graft-versus-Host Disease that Persists Despite Steroids

Full Title

A Single-arm, Prospective Study of Remestemcel-L, Ex-vivo Cultured Adult Human Mesenchymal Stromal Cells, for the Treatment of Pediatric Patients who Have Failed to Respond to Steroid Treatment for Acute GVHD


Patients who have a stem cell transplant can experience a complication called graft-versus-host disease (GVHD), in which cells from the donor attack tissues of the recipient. Symptoms of GVHD include rash, digestive problems, weight loss, and liver dysfunction. GVHD is usually treated with steroid drugs (corticosteroids), but patients may experience symptoms during or after treatment.

In this study, researchers are evaluating the use of mesenchymal stem cells (MSC or remestemcel-L) in children with acute GVHD affecting the skin, liver, and/or digestive tract. MSC come from the bone marrow and produce some of the body’s tissues, such as bone, cartilage, tendons, muscle and fat. Studies suggest MSC may reduce the immune system activity causing GHVD, and can find damaged tissue and help repair it. Remestemcel-L is given intravenously (by vein).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must be between the ages of 2 months and 17 years (inclusive).
  • Patients must have acute GVHD that did not get better with steroid therapy.

For more information about this study and to inquire about eligibility, please contact Dr. Susan Prockop at 212-639-6715.





Disease Status

Newly Diagnosed & Relapsed/Refractory