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SCIDBMT: Partial-Matched Bone Marrow Transplant for Infants with Severe Combined Immunodeficiency (SCID)

About this study

Severe combined immunodeficiency (SCID) is a disease that affects the immune system. It is commonly known as “bubble boy” disease. Children with SCID are more likely to get infections because their immune systems do not work properly.

Hematopoietic cell transplant (HCT) is a common procedure used to treat SCID and other diseases. HCT is also known as stem cell transplant or bone marrow transplant.

HCT begins with anti-cancer medicine called chemotherapy. This medicine damages or kills the bone marrow to make space within the bones for new blood cells. Next, doctors give your child healthy blood-making cells from a donor. These cells travel through the blood to the bone marrow space, where they begin to grow and make new blood cells.

The best donor for a bone marrow transplant is a brother or sister who is a “match” for your child’s immune type. If your child does not have a sibling match, someone who is a close or partial match may donate blood cells. This donor might be another family member or an unrelated volunteer.

In this study, doctors will give your child immune cells from an unrelated donor or family member to provide some immune protection before your child can make his/her own immune cells. If your child has a matched sibling, no additional immune cells will be given.

Eligibility overview

  • Infant at least 2 months old with SCID diagnosis
  • Has a suitable matched sibling donor or matched unrelated donor, or a single haplotype matched family member donor

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full title:

Haplocompatible Transplant Using TCRa/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)

Study goal:

The main goal of this study is to learn more about the good and bad effects of this procedure. The study will look at how bone marrow transplant from a partial-matched donor affects a child’s disease and overall survival.

Age:

Infants, at least 2 months old

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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