A Phase I, Open-label, Multi-center Study of Clofarabine in Japanese Paediatric Patients With Relapsed or Refractory Acute Lymphoblastic Leukaemia
This is a Phase I Study of Clofarabine in Japanese Paediatric Patients With Relapsed or
Refractory Acute Lymphoblastic Leukaemia.
Subjects will receive intravenous administration of clofarabine at 30 or 52 mg/m2/day (2
hours) for 5 consecutive days and then the administration will be withheld until Day 14. If
there is no evidence of recovery in neutrophil (≥750/mm3) and/or platelet count
(≥50,000/mm3), the therapy may be withheld up to Day 42. However, in the absence of
progression based on the judgment of the investigator after each cycle of treatment and the
benefit of continued treatment is judged to exceed the risk, subjects may receive up to a
total of six cycles. If a subject is receiving two or more cycles, a written consent must
be obtained prior to start of Cycle 2.
When a subject completes the final dose, the safety will be observed and followed-up for 45
days after the final study drug administration.
Cohort 1 will receive 30 mg/m2/day x 5 days at Cycle 1 and will be assessed for
tolerability. Samples will be drawn to assess pharmacokinetics at this dose. If subjects
do not develop adverse events indicative of dose limiting toxicity (DLT) at Cycle 1, the
dose will be increased to 52 mg/m2/day x 5 days from Cycle 2 and the subjects will be
assessed for safety and activity only.
Cohort 2 will receive 52 mg/m2/day x 5 days at Cycle 1 and will be assessed for the
tolerability. Samples will be drawn to assess pharmacokinetics at this dose.
Whether or not proceeding to Cohort 2 after the Cycle 1 of Cohort 1 is completed will be
determined by the sponsor based on the assessment of the safety data and the recommendation
of the Data Safety Monitoring Board (DSMB).
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum Tolerated Dose (MTD)
14 days (1st cycle)
Japan: Ministry of Health, Labor and Welfare