Clinical trial NCT00354419 (phase 1, status: terminated)
Conditions: aplastic anemia
ICD codes: D61.3, D61.9, D61.09, D61.1, D61.2, D61.89
Drugs: cyclophosphamide, cyclosporine, mycophenolate mofetil
SMILES: ClCCN(CCCl)P1(=O)NCCCO1, CC[C@@H]1NC(=O)[C@H]([C@H](O)[C@H](C)C\C=C\C)N(C)C(=O)[C@H](C(C)C)N(C)C(=O)[C@H](CC(C)C)N(C)C(=O)[C@H](CC(C)C)N(C)C(=O)[C@@H](C)NC(=O)[C@H](C)NC(=O)[C@H](CC(C)C)N(C)C(=O)[C@@H](NC(=O)[C@H](CC(C)C)N(C)C(=O)CN(C)C1=O)C(C)C, N[C@@H](CCCNC(N)=N)C(O)=O
Reason stopped: 
    low accrual
  
Eligibility criteria:

        Inclusion Criteria:

          -  Life-threatening marrow failure of nonmalignant etiology meeting two of the three
             following criteria: granulocytes < 500/mm^3; a corrected reticulocyte count < 1%;
             platelet count < 20,000/mm^3

          -  Failure to respond to the best available immunosuppressive treatment protocol by 75
             days after initiation of therapy

          -  Lack of an HLA-identical family member or closely matched (9 or 10 of 10 HLA-locus
             match) unrelated marrow donor

          -  DONOR: Unrelated UCB unit matched for at least 4 of 6 loci

          -  DONOR: Related UCB unit matched for at least 3 of 6 lock

          -  Selection of the UCB unit(s) will be based upon matching or mismatching at HLA-A, B
             antigen level and DRB1 allele level typing; while HLA-C antigen/allele and HLA-DQB1
             antigen/allele level typing are not considered in the matching criteria, if available
             each may be used to optimize unit selection

          -  Multiple UCB units are allowed to provide sufficient cell dose; when multiple units
             are selected, the following rules apply: a) the UCB unit with the least HLA disparity
             will be selected first (i.e., selection priority is 6/6 match > 5/6 match > 4/6
             match), additional UCB units may be selected to increase cell dose; b) UCB units must
             be matched to each other for at least 4 of 6 loci; c) each unit must contain at least
             1.5 x 10^7 Total Nucleated Cells per kg recipient weight; d) the total cell dose of
             the combined units must be at least 3.0 x 10^7 Total Nucleated Cells per kg recipient
             weight

        Exclusion Criteria:

          -  Severe disease other than aplastic anemia that would severely limit the probability of
             survival during the graft procedure; patients who present with active fungal
             infections must be treated to resolve this problem before beginning the conditioning
             regimen

          -  HIV seropositive patients

          -  Patients who have developed clonal cytogenetic abnormalities or a myelodysplastic
             syndrome (these patients will be considered in separate protocols for myelodysplastic
             syndrome, etc.)

          -  Patients with paroxysmal nocturnal hemoglobinuria or Fanconi anemia

          -  Patients > 40 years of age

          -  Related or unrelated cord blood units with < 1.5 x 10^7 Total Nucleated Cells per kg
             recipient weight

          -  Related or unrelated cord blood units without full testing and negative results for
             hepatitis A, B, C, HIV, HTLV-1, CMV viruses