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Protocol No. UW19015

Principal Investigator Hofmann, Inga

Phase III

Age Group Children

Scope National

Sponsor Type Externally Peer-Reviewed

Title Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia (TransIT) PBMTC NMD1601

Objective The goal of this study is to randomize patients to either a matched unrelated donor transplant versus immune therapy. The purpose of this study is to use this approach at a small number of centers before expanding the trial to a much larger number of centers. This larger study will determine which therapy is the best for younger patients at the time of diagnosis of SAA

Treatment If you choose to participate in this research study you will be randomly assigned to receive immunosuppressive therapy (IST) or a matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT). Immune therapy is current standard therapy
IST Arm: ATGAM (horse ATG) 40 mg/kg IV for 4 days and cyclosporine IV or PO started on the first day of treatment and administered for a minimum of 12 months
HSCT Arm: Preparative regimen will be FACT, which consists of fludarabine 120 mg/m2 , ATG 9 mg/kg,cyclophosphamide 50mg/kg, and TBI 200 cGY with cyclosporine and methotrexate GVHD prophylaxis

Description Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

Key Eligibility Inclusion Criteria:
  • Confirmed diagnosis of idiopathic SAA, Age less than or equal to 25 years of age, and no suitable fully matched related donor available
  • At least 2 unrelated donors noted on NMDP search who are well matched (9/10 or 10/10 for HLA-A, B, C, DRB1, and DQB1 using high resolution)
  • Adequate organ function
    HSCT Arm Inclusion Criteria:
  • Patient randomized to the HSCT arm
  • Unrelated donor who is 9/10 or 10/10 match for HLA-A, B, C, DRB1, and DQB1 at high resolution typing using DNA-based typing confirmed and cleared
  • Signed informed consent for HSCT
  • Karnofsky or Lansky performance status greater than or equal to 60%
  • Adequate organ function
    Exclusion Criteria:
  • Inherited bone marrow failure syndromes (IBMFS)
  • Myelodysplastic syndrome (MDS)
  • Known severe allergy to horse ATG
  • Prior allogeneic stem cell transplant
  • Prior solid organ transplant
  • Infection with human immunodeficiency virus (HIV)
  • Active Hepatitis B or C
  • Female patients who are pregnant or breast-feeding
  • Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in sit
    HSCT Arm Exclusion Criteria: Uncontrolled, progressive infection

  • Applicable Disease Sites Hematologic cancers, other

    Status Open

    Participating Institutions UW Hospital and Clinics