Pediatric Bone Marrow Transplantation Clinical Trials

At MSK Kids, your child may be able to take part in a clinical trial, also known as a clinical research study. In a clinical trial, doctors evaluate a new therapy or treatment combination to find out whether it is more-effective than current treatments. Nearly every cancer treatment used today exists because of a clinical trial. For every study, the goal is the same: to cure the disease and enable the patient to live a long, healthy life.

Our patients have access to many of the latest clinical trials, some of which are available only at MSK. For example, studies are evaluating new approaches to T-cell depletion that reduce the risk of graft-versus-host-disease while maximizing the immune system’s ability to recover after a transplant. Researchers are also looking at modified T cells and new medications to treat viral infections after transplantation. A new nontoxic regimen to prepare a child for a bone marrow transplant is also being studied. It uses an antibody instead of chemotherapy and radiation therapy before the transplant, which may reduce short- and long-term side effects in your child.

MSK Kids also takes part in many nationwide clinical trials as a founding member of the Children’s Oncology Group, the coordinating center and co-founder of the Pediatric Oncology Experimental Therapeutics Investigators’ Consortium, and through other multicenter trial networks across the United States and around the world.

Your child’s care team will let you know if your child can participate in a clinical trial. The decision to enroll is entirely voluntary. The study team will explain the benefits and risks of the study to you so you can make an informed decision.

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7 Clinical Trials found
Chronic graft-versus-host disease (cGVHD) is a condition in which healthy transplanted stem cells attack the recipient's healthy cells. cGVHD most often happens more than 100 days after a stem cell transplant, but it can happen at any time.
Primary immune regulatory disorder (PIRD) and autoinflammatory conditions are conditions of the immune system that can cause an unusual amount of inflammation. While a stem cell transplant is a standard treatment for people with a PIRD or autoinflammatory condition, the inflammation caused by these conditions can reduce the effectiveness of this treatment.
Researchers want to see if a steroid-free approach using ruxolitinib, with or without axatilimab, works well to treat cGVHD. Ruxolitinib blocks JAK proteins, which may reduce inflammation (swelling) and immune system reactions related to GVHD. Axatilimab blocks a protein called CSF-1R that controls white blood cells which play a role in GVHD.
Severe aplastic anemia is a serious condition where the bone marrow does not make the normal amount of blood cells. Bone marrow transplantation is one way to treat this disease. It has been reserved for people under age 40 with a related donor whose cells fully match theirs. However, advances have been made in bone marrow transplantation that make it an option for more people.
The purpose of this study is to assess the safety and effectiveness of a specialized light treatment for children and adults with chronic graft-versus host disease (GVHD) of the mouth (oral GVHD) that has not improved after standard treatment. GVHD is a complication that can occur after a stem cell or bone marrow transplant. The newly transplanted donor cells attack the transplant recipient's body and cause serious health problems. Oral GVHD can cause mouth inflammation, pain, and sores.
The transplantation of stem cells from umbilical cord blood is a treatment for some blood cancers and non-cancerous blood or metabolic disorders. Patients routinely receive high doses of chemotherapy and sometimes radiation before receiving the stem cells to help make room in the bone marrow for new blood stem cells to grow, prevent the body from rejecting the transplanted cells, and help kill any abnormal blood cells in the body. However, the combination of these treatments can have serious side effects.
Sickle cell disease and beta-thalassemia are blood diseases caused by a genetic change (mutation) in hemoglobin, the protein in red blood cells that carries oxygen throughout the body. People with these diseases may be offered a stem cell transplant. Stem cell transplantation involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in the bone marrow.