A Phase II Study to See How Different Doses of Busulfan Help Improve the Immune System in Infants with Severe Combined Immunodeficiency (SCID) Having a Stem Cell Transplant

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Full Title

A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRaß+/CD19+ Depleted Transplantation: A Phase II Study By The Primary Immune Deficiency Treatment Consortium (PIDTC) And Pediatric Blood And Marrow Transplant Consortium (PBMTC) PIDTC “CSIDE” Protocol (Conditioning SCID Infants Diagnosed Early) PBMTC NMD 1801(NMDP IRB)

Purpose

Severe combined immunodeficiency (SCID) is a genetic disorder of the immune system. Children with SCID have very little immune function and can’t fight off infections. Children with certain types of SCID can achieve good function in their T cells (a type of white blood cell) after transplant without chemotherapy, but usually have poor B-cell function (another immune cell) after transplant. However, chemotherapy causes side effects.

In this study, researchers want to see if low and medium doses of a chemotherapy drug called busulfan (given in addition to other drugs) will help babies with SCID achieve good immunity with few complications after transplantation. Infants with SCID who are most likely to benefit from this approach and who have unrelated or mismatched stem cell donors will receive a transplant early in life, before they get severe infections, or later if their infections are under control.

Eligibility

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

  • Patients must be babies age newborn to 2 years with SCID (typical or “leaky” SCID or Omenn syndrome).
  • This study is for patients without a related stem cell donor. They may have an unrelated or mismatched donor.

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

Protocol

20-016

Phase

II

Disease Status

Newly Diagnosed

Investigator

Co-Investigators