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Clinical Trial 19089

Cancer Type:
Interventions:Thymoglobulin; cyclophosphamide; cytoxan (cyclophosphamide); fludarabine (Fludarabine phosphate)

Study Type: Treatment
Phase of Study: Phase II
Investigators:

  • Lia Perez

Overview

Study Title

Optimizing Cord Blood and Haploidentical Aplastic Anemia Transplantation (CHAMP)

Summary

The purpose of this study is to learn how well severe aplastic anemia (SAA) participants do with transplants that use unrelated cord blood and transplants that use haploidentical bone marrow. Moffitt Cancer Center will only enroll participants with a haploidentical bone marrow donor.

Objective

Assess overall survival (OS) separately in 2 cohorts (unrelated cord blood [UCB] and haploidentical [haplo] marrow) at 1 year post-hematopoietic stem cell transplantation (HSCT) in patients with severe aplastic anemia (SAA). Assess overall survival (OS) separately in 2 cohorts (unrelated cord blood [UCB] and haploidentical [haplo] marrow) at 1 year post-hematopoietic stem cell transplantation (HSCT) in patients with severe aplastic anemia (SAA).

Inclusion Criteria

  • Less than 75 years of age at time of enrollment.
  • Confirmed diagnosis severe aplastic anemia (SAA), either from initial diagnosis or follow-up assessments, defined as: Bone marrow cellularity less than 25% or marrow cellularity less than 50% but with less than 30% residual hematopoietic cells. Two out of three of the following (in peripheral blood): Neutrophils less than 0.5 x10^9/L, Platelets less than 20 x10^9/L, or Reticulocyte count less than> No suitable fully matched related or unrelated donor available. Search for an unrelated donor and enrollment on this protocol may be abandoned if the clinical situation dictates an urgent transplant in the best medical judgment of the treating provider. The definition of clinical urgency may include a low likelihood of identifying a suitable matched unrelated donor within 6-8 weeks from referral and the medical need to choose a donor without further delay beyond that time.
  • Failed at least one trial of immunosuppressive therapy (IST) by being refractory or having relapsed. IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA.
  • Available alternative donor: Human Lymphocyte Antigen (HLA) haploidentical (haplo) first degree relatives of the patient including biological parents, siblings or half siblings, or children with 2, 3, or 4 mismatches using DNA-based typing. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch. The donor and recipient must be identical at a minimum of one allele (at high resolution DNA-based typing) at the following genetic loci: HLA-A, -B, -C, and DRB1.
  • Patient and/or legal guardian must sign informed consent for Hematopoietic Stem Cell Transplantation (HSCT).
  • In the haplo cohort, the donor and/or legal guardian must be able to sign informed consent documents.
  • In the haplo cohort, the potential donor must be willing to donate bone marrow.
  • In the haplo cohort, the weight of the haplo donor must be ≥ 20 kg.
  • Must have adequate cardiac, hepatic, renal and pulmonary organ function.
  • Karnofsky or Lansky performance status ≥ 60%.
  • Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence.
  • Additional criteria may apply.

    • Exclusion Criteria

  • Inherited bone marrow failure syndromes. At minimum, the diagnosis of Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on peripheral blood or marrow.
  • Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (e.g., Monosomy 7).
  • Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement-dependent cytotoxicity or flow cytometric testing or the presence of anti-donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) > 1000 by solid phase immunoassay).
  • Prior allogeneic stem cell transplant.
  • Prior solid organ transplant.
  • Known life-threatening reaction (i.e., anaphylaxis) to Thymoglobulin® that would prohibit use for the patient as this study requires use of the Thymoglobulin® preparation of ATG.
  • Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment.
  • Seropositive for the human immunodeficiency virus (HIV).
  • Active Hepatitis B or C determined by a detectable viral load of HBV or HCV.
  • Female patients who are pregnant (per institutional practice) or breast-feeding.
  • Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent > 5 years previously will be allowed. Cancer treated with curative intent less than or equal to 5 years previously will not be allowed unless approved by the Protocol Chairs and/or Protocol Officer.
  • Alemtuzumab or Antithymocyte globulin (ATG) within 2 weeks of enrollment.

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