Clinical trial NCT01295307 (phase 2, status: completed)
Conditions: acute myeloid leukemia
ICD codes: C92.A1, C92.A2, C92.61, C92.62, C92.A0, C92.60
Drugs: clofarabine
SMILES: [H][C@@]12C[C@@H](C)[C@](O)(C(=O)CO)[C@@]1(C)C[C@H](O)[C@@]1(F)[C@@]2([H])CCC2=CC(=O)C=C[C@]12C
Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of AML according to WHO criteria.

          -  Untreated relapse or refractory disease after a minimum of one standard induction
             therapy. Treatment of relapse with leukocyte-apheresis or up to 5 days with low dose
             cytarabine or hydroxyurea is allowed.

               -  Refractory disease is defined as ≥5% blasts after the second cycle of induction
                  therapy or no reduction in marrow blasts at early treatment assessment (day +15)
                  after the first cycle of induction therapy.

               -  Relapse is defined as an increase in bone marrow blast count ≥5%, re-appearance
                  of blasts in the peripheral blood or extramedullary disease.

          -  Age above 40 years.

          -  Have adequate renal and hepatic functions as indicated by the following laboratory
             values:

               -  Serum creatinine <=1.0 mg/dL; if serum creatinine >1.0 mg/dL, then the estimated
                  glomerular filtration rate (GFR) must be >60 mL/min/1.73 m2 (see reference
                  below*)

               -  Serum bilirubin <=1.5× upper limit of normal (ULN)

               -  Aspartate transaminase (AST)/alanine transaminase (ALT) <=2.5× ULN

               -  Alkaline phosphatase <=2.5× ULN

          -  Eligibility for intensive chemotherapy

          -  Patient needs to be capable to understand the clinical trial as an investigational
             approach to bridge the time to potential allogeneic HCT, potential risks and benefits
             of the study.

          -  Signed written informed consent.

          -  Female patients of childbearing potential must have a negative serum

          -  Male and female patients must use an effective contraceptive method during the study
             and for a minimum of 6 months after study treatment.

        Exclusion Criteria:

          -  For refractory disease, more than two prior induction chemotherapies or more than one
             prior salvage chemotherapy containing high-dose cytarabine (cumulative dose of
             cytarabine ≥ 5 g/m2).

          -  Second or higher relapse. Patients who received hypomethylating agents like
             azacytidine or decitabine as a treatment of first relapse, respond and relapse later
             on may be included.

          -  Acute promyelocytic leukemia with t(15;17)(q22;q12) molecular detection or (PML/RARα).

          -  Central nervous system involvement (i.e. WBC ≥ 5/µL in cerebrospinal fluid with blasts
             present on cytospin).

          -  Prior allogeneic HCT

          -  Autologous transplantation within 100 days prior to start of study treatment

          -  Use of investigational agents or anticancer therapy within 10 days before study entry
             with the exception of hydroxyurea or low-dose cytarabine.

          -  Have any other severe concurrent disease, or have a history of serious organ
             dysfunction or disease involving the heart, kidney, liver, or other organ system that
             may place the patient at undue risk to undergo transplantation.

          -  Patients with known refractoriness to platelet support.

          -  Patients with a systemic fungal, bacterial, viral, or other infection not controlled
             (defined as exhibiting ongoing signs/symptoms related to the infection and without
             improvement, despite appropriate antibiotics or other treatment).

          -  Pregnant or lactating patients.

          -  Any significant concurrent disease, illness, or psychiatric disorder that would
             compromise patient safety or compliance, interfere with consent, study participation,
             follow up, or interpretation of study results.