for people ages up to 75 years (full criteria)
at Oakland, California and other locations
study started
estimated completion:



This study is a prospective, simultaneous, parallel phase II study with one arm receiving unrelated cord blood transplantation and the other arm receiving haploidentical transplantation for Severe Aplastic Anemia (SAA) patients. The primary objective is to assess overall survival (OS) separately in the 2 arms at 1 year post-hematopoietic stem cell transplantation (HSCT).

Official Title

Optimizing Cord Blood and Haploidentical Aplastic Anemia Transplantation (CHAMP) (BMT CTN #1502)


Acquired SAA is a rare bone marrow failure disorder with an estimated annual incidence of 2 cases per million and with over 600 new cases in the United States each year. A major challenge in treating acquired SAA is the management of patients who are refractory to immunosuppressant therapy (IST) or have relapsed after IST. HSCT is the only curative option for these patients but many are ineligible because they lack a suitable donor. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) seeks to increase options for these patients by using novel therapeutic strategies (optimized preparative regimens and targeted antithymocyte globulin (ATG) pharmacokinetics (PK)) to expand the donor pool utilizing UCB for transplantation. The goal of this protocol is to test whether optimized approaches using two alternative donor sources will achieve acceptable outcomes in SAA patients. Ideally, a direct comparison of the two approaches would allow us to determine a best approach. However, given 1) the rarity of the disease, 2) the fact that different centers often prefer different stem cell sources, and 3) the fact that most comparisons of alternative donor stem cell sources have shown very similar outcomes, this protocol allows both approaches to be performed, but there are no planned comparisons of outcomes between the two cohorts.


Severe Aplastic Anemia Unrelated Cord Blood (UCB) Haploidentical Bone Marrow Hematopoietic Stem Cell Transplant (HSCT) Severe Aplastic Anemia (SAA) Antithymocyte Globulin (ATG) Anemia Anemia, Aplastic Cyclophosphamide Tacrolimus Fludarabine phosphate Thymoglobulin Antilymphocyte Serum Fludarabine Mycophenolic Acid Vidarabine Antithymocyte Globulin Total Body Irradiation (TBI) UCB HSCT Haplo HSCT Mycophenolate mofetil (MMF) G-CSF Unrelated Cord Blood HSCT Haplo Bone Marrow HSCT


You can join if…

Open to people ages up to 75 years

  1. Patient is < 75 years of age at time of enrollment.
  2. Confirmed diagnosis of SAA, either from initial diagnosis or follow-up assessments,defined as:
  3. Bone marrow cellularity < 25% or marrow cellularity < 50% but with < 30% residual hematopoietic cells.
  4. Two out of three of the following (in peripheral blood): Neutrophils < 0.5 x109/L, Platelets < 20 x109/L, or Reticulocyte count < 20 x109/L

  5. No suitable fully matched related (6/6 match for human leukocyte antigen (HLA)-A and B at intermediate or high resolution and DRB1 at high resolution using DNA-based typing)or unrelated donor (8/8 match for HLA-A, B, C, and DRB1 at high resolution using DNA-based typing) 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.
  6. 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.
  7. Available alternative donor:
  8. Cord blood unit(s) must be matched at a minimum of 4/6 to the recipient at HLA-A and B at low resolution using DNA-based typing and HLA-DRB1 at high resolution using DNA-based typing. Based on a published report from Eurocord, for single unit transplantation, the single unit pre-cryopreserved total nucleated cell(TNC) dose must be a minimum of 4.0 x107/kg recipient weight. For double cord transplants, each unit must have a minimum of 1.5 x107/kg pre-cryopreserved TNC and a minimum total of 4.0 x107/kg (sum of unit 1 and unit 2). For non-red blood cell depleted units, the minimum pre-cryopreserved TNC dose is 2.0 x107/kg recipient weight.


  1. HLA 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.
  2. Patient and/or legal guardian must sign informed consent for HSCT.
  3. In the haplo cohort, the donor and/or legal guardian must be able to sign informed consent documents.
  4. In the haplo cohort, the potential donor must be willing to donate bone marrow.
  5. In the haplo cohort, the weight of the haplo donor must be ≥ 20 kg.
  6. . Adequate organ function defined as:
  7. Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40%. For patients aged < 13 years, shortening fraction (SF) ≥ 26% by echocardiogram or Multi Gated Acquisition Scan (MUGA) may be substituted for LVEF.
  8. Hepatic: Total bilirubin < 3.0 x the upper limit of normal (ULN) for age(patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and alanine aminotransferase (ALT) and aspartate aminotransferase(AST) < 5.0 x ULN for age.
  9. Renal: For patients > 13.0 years of age at the time of enrollment: estimated creatinine clearance > 50 mL/minute (using the Cockcroft-Gault formula and actual body weight). For patients < 13.0 years of age at enrollment: Glomerular Filtration Rate (GFR) estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2.

  10. Pulmonary: For patients > 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO) (corrected/adjusted for hemoglobin) > 40% and forced expiratory volume in one second (FEV1) > 50% predicted (without administration of bronchodilator) and forced vital capacity (FVC) > 50% predicted. For patients <13.0 years of age unable to perform pulmonary function tests (PFT) due to age or developmental ability: (1) no evidence of dyspnea at rest and (2) no need for supplemental oxygen and (3) O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care(e.g., Utah, 4,200 feet above sea level, does not give supplemental oxygen unless below 90%)).
  11. . Karnofsky or Lansky performance status ≥ 60%.
  12. . Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence.

You CAN'T join if...

  1. 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.
  2. Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome(pre-MDS) or MDS on marrow examination (e.g. Monosomy 7).
  3. 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).
  4. Prior allogeneic stem cell transplant.
  5. Prior solid organ transplant.
  6. 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.
  7. 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.
  8. Seropositive for the human immunodeficiency virus (HIV).
  9. Active Hepatitis B or C determined by a detectable viral load of HBV or HCV.
  10. . Female patients who are pregnant (per institutional practice) or breast-feeding.
  11. . 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 ≤ 5 years previously will not be allowed unless approved by the Protocol Chairs and/or Protocol Officer.
  12. . Alemtuzumab or ATG within 2 weeks of enrollment.


  • Children's Hospital at Oakland not yet accepting patients
    Oakland California 94609 United States
  • Stanford Hospital and Clinics not yet accepting patients
    Stanford California 94305 United States


accepting new patients
Start Date
Completion Date
Medical College of Wisconsin
Blood and Marrow Transplant Clinical Trials Network Website
National Marrow Donor Program
Phase 2
Study Type
Last Updated
August 29, 2017