Pediatric Blood & Marrow Transplant (BMT)

Clinical Trials

Clinical trials at Johns Hopkins All Children’s Hospital provide bone and marrow transplant patients access to research opportunities to advance the diagnosis, treatment and prevention of pediatric-onset diseases.

Patients who receive a blood or marrow transplant at Johns Hopkins All Children’s Hospital may be asked to participate in a clinical trial. A clinical trial is a research study that is designed to help determine the best treatment for a patient with a disease. These trials help us further our understanding of treatments and diseases, helping us improve care for children with cancer and blood disorders.

Participating in a clinical trial is optional, but may benefit children with similar conditions. If the patient or the patient’s parent chooses to participate, the doctor and the consent document will provide detailed information on the treatment and its potential risks and benefits.

These clinical trials are sponsored by Johns Hopkins All Children’s Blood & Marrow Transplant Program and the adult transplant program at the H. Lee Moffitt Cancer Center. The transplant teams meet weekly to share resources. Additional clinical trials are sponsored by the Pediatric Blood and Marrow Transplant Consortium and the Children’s Oncology Group.

What is a Clinical Trial?

A clinical trial outlines a specific treatment plan. It may include specific chemotherapy drugs or supportive care therapies such as medicines to prevent nausea, vomiting, infections or other complications of transplantation. Each clinical trial has a protocol that explains how it will work and must be approved by the organization sponsoring the study.

Cancer clinical trials include a three-phase research process that answers different questions about a new treatment:

  • Phase 1: The purpose of phase 1 is to determine a safe dosage for the patient, decide how the treatment should be given (by mouth, vein, etc.) and to see how the treatment affects the body and fights cancer.
  • Phase 2: Phase 2 determines if the new treatment fights the cancer.
  • Phase 3: In phase 3, results of patients taking the new treatment are compared with patients taking the standard treatment.

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