Clinical trial NCT02433483 (phase 2, status: terminated)
Conditions: acute myeloid leukemia (aml), myelodysplastic syndrome (mds)
ICD codes: C95.91, C95.92, Z80.6, Z85.6, C90.11, C90.12, C91.01, D46.Z, D46.9, C94.6, D46.C
Drugs: cytarabine, intrathecal triples
SMILES: ClCCN(CCCl)P1(=O)NCCCO1, CN(CC1=CN=C2N=C(N)N=C(N)C2=N1)C1=CC=C(C=C1)C(=O)N[C@@H](CCC(O)=O)C(O)=O
Reason stopped: 
    the study was closed due to poor accrual and because of competing protocols.
  
Eligibility criteria:

        INCLUSION CRITERIA - AML and MDS PARTICIPANTS

          -  Participants must have a diagnosis of AML or myelodysplastic syndrome (MDS), ALL, and
             must have disease that has relapsed or is refractory to chemotherapy, or that has
             relapsed after HSCT.

               -  Refractory disease is defined as persistent disease after at least two courses of
                  induction chemotherapy.

               -  Patients with AML must have ≥ 5% leukemic blasts in the bone marrow or have
                  converted from negative minimal residual disease (MRD) status to positive MRD
                  status in the bone marrow as assessed by flow cytometry. If an adequate bone
                  marrow sample cannot be obtained, patients may be enrolled if there is
                  unequivocal evidence of leukemia in the peripheral blood.

          -  Participant is ≤ 21 years of age (i.e., has not reached 22nd birthday).

          -  Adequate organ function defined as the following:

               -  Total bilirubin ≤ upper limit of normal (ULN) for age, or if total bilirubin is >
                  ULN, direct bilirubin is ≤ 1.5 mg/dL

               -  AST (SGOT)/ALT (SGPT) < 5 x ULN

               -  Calculated creatinine clearance > 50 ml/min/1.73m^2 as calculated by the Schwartz
                  formula for estimated glomerular filtration rate >

               -  Left ventricular ejection fraction ≥ 40% or shortening fraction ≥ 25%.

          -  Has an available HPC-A donor.

          -  Performance status: Lansky ≥ 50 for patients who are ≤ 16 years old and Karnofsky ≥
             50% for patients who are > 16 years old.

          -  Does not have an uncontrolled infection requiring parenteral antibiotics, antivirals,
             or antifungals within one week prior to first dose. Infections controlled on
             concurrent anti-microbial agents are acceptable, and anti-microbial prophylaxis per
             institutional guidelines is acceptable.

          -  Patient has fully recovered from the acute effects of all prior therapy and must meet
             the following criteria.

               -  At least 14 days must have elapsed since the completion of myelosuppressive
                  therapy.

               -  At least 24 hours must have elapsed since the completion of hydroxyurea, low-dose
                  cytarabine (up to 200 mg/m^2/day), and intrathecal chemotherapy.

               -  At least 30 days must have elapsed since the use of investigational agents.

               -  For patients who have received prior HSCT, there can be no evidence of GVHD and
                  greater than 60 days must have elapsed since the HSCT. Patients cannot be
                  receiving therapy, including steroids, for GVHD.

          -  Post-menarchal female has had negative serum pregnancy test within 7 days prior to
             enrollment.

          -  Male or female of reproductive potential has agreed to use effective contraception for
             the duration of study participation.

          -  Not breastfeeding

        INCLUSION CRITERIA - HPC-A CELL DONOR

          -  At least 18 years of age.

          -  Family member (first degree relatives).

          -  Not pregnant as confirmed by negative serum or urine pregnancy test within 7 days
             prior to enrollment (if female).

          -  Not breast feeding.

          -  Meets donation eligibility requirements as outlined by 21 CFR 1271.