Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
Status:
Active, not recruiting
Trial end date:
2025-01-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to determine the safety and effectiveness of Umbilical Cord
Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can
decrease the incidence of GVHD.
This study is for patients that were born with a disease that affects their body's metabolism
or immune system. The doctor plans to treat the patient for this illness with a stem cell
transplant.
While improved medical care has allowed many people with these diseases to live longer, the
only way to truly cure the diseases is by means of a stem cell transplant from a donor who
does not have the disease. A stem cell transplant will replace sick cells with new healthy
donor cells. Stem cells grow into different types of blood cells that people need, including
red blood cells, white blood cells, and platelets. In a stem cell transplant, the patients
own stem cells would be killed by chemotherapy drug and then replaced by stem cells from the
donor. Stem cells can be collected from the bone marrow, peripheral blood or umbilical cords.
In this study, umbilical cords will be the source of the stem cells.
Currently, large inventories of umbilical cord blood units are available in public banks for
transplantation in those lacking bone marrow donors. UCB transplants offer several advantages
over adult bone marrow or peripheral blood stem cell transplants, including:
1. Rapid availability,
2. Absence of donor risk,
3. Low risk of transmissible infectious diseases,
4. Low risk of acute GvHD (as compared to recipients of unrelated donor marrow and
peripheral blood cells).
The two main causes of death after umbilical cord blood transplantation for disorders for
these kinds of patients, are graft failure and infection.
In this study we are trying to address these two problems by using different drugs to prepare
patients for the transplant.
To help improve engraftment (cells begin to grow), we will include the drug Fludarabine to
the usually used Busulfan and Cytoxan that the study patients will receive before their
transplant.
We will try to decrease the chance of developing graft-versus-host disease (GvHD) by using
Cyclosporin A (CSA) and Mycophenolate Mofetil (MMF), instead of Anti-Thymocyte Globulin (ATG)
which is normally used.
Phase:
N/A
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Center for Cell and Gene Therapy, Baylor College of Medicine Texas Children's Hospital