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Protocol No. UW18143

Principal Investigator DeSantes, Kenneth

Phase II

Age Group Children

Scope National

Sponsor Type Externally Peer-Reviewed

Title A Randomized Trial of Low vs. Moderate Exposure Busulfan for Infants with Severe Combined Immunodeficiency (SCID) Receiving TCR alpha beta+/CD19+ Depleted Transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC)

Objective The purpose of this study is to see if lower doses of a chemotherapy drug called busulfan will help babies with certain types of SCID achieve good immunity after receiving a hematopoietic stem cell transplant (HSCT) that has some immune cells (called T cells) removed

Treatment
  • Your child will receive either a low or medium dose of the chemotherapy drug busulfan, determined by randomization (selection by chance)
  • Your child will receive donor stem cells that have been modified to remove most of the T-cell and B-cells
  • A cheek swab (also called a buccal swab) will be taken from your child prior to the transplant to determine the percentage of donor cells versus your child’s cells in your child’s blood after transplant
  • Research blood study samples to learn more about SCID and how blood cells help the immune system will be collected before transplant, the day of transplant, and at 7 days, 14 days, 42 days, 100 days, 6 months, 9 months, 1 year, 2 years, and possibly 3 years post-transplant
  • Approximately 9-18 months after transplant, vaccinations will be administered, and a blood test measuring whether your child’s body has responded to the vaccine will be collected

  • Description Phase II Conditioning SCID Infants Diagnosed Early (CSIDE)

    Key Eligibility
  • Infants with SCID, either typical or leaky or Omenn syndrome
  • Typical SCID is defined as either of the following: Absence or very low number of T cells (CD3+ T cells less than 300/microliter AND no or very low T cell function (less than 10% of lower limit of normal) as measured by response to phytohemagglutinin OR Presence of maternally derived T cells
  • Leaky SCID is defined as the following: Absence of maternally derived T cells AND either one or both of the following (i, ii): less than 50% of lower limit of normal T cell function as measured by response to PHA OR < less than 30% of lower limit of normal T cell function as measured by response to CD3 Absent or less than 10% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens (must document post vaccination or exposure for this criterion to apply)
  • AND at least two of the following (i through iii): i. CD3 T cells less than 1500/microliter, ii. greater than 80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR less than 80% of CD3+ or CD4+ T cells are CD62L negative AND/OR greater than 50% of CD3+ or CD4+ T cells express HLA-DR (at less than 4 years of age) AND/OR are oligoclonal T iii. Low TRECs and/or the percentage of D4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower level of normal
  • Omenn syndrome: as defined by the protocol
  • Documented mutation in one of the following SCID-related genes: as defined by the protocol
  • No available genotypically matched related donor (sibling)
  • Availability of a suitable donor and graft source as defined by the protocol
  • Age 0 to 2 years at enrollment
  • Adequate organ function as defined by the protocol
    EXCLUSION
  • Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen as defined by the protocol
  • Patients with HIV or HTLV I/II infection will be excluded

  • Applicable Disease Sites Hematologic cancers, other

    Status Open

    Participating Institutions UW Hospital and Clinics