Philadelphia chromosome positive leukemia relapsed/refractory
Conditions
Interventions
oral Ponatinib at a dose of 15~25 mg/m^2 for 28 days.
Sponsors
Kodama Yuichi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Diagnosis of CML that was resistant/intolerant to 2nd generation TKI, or of Ph+ALL that was relapsed/refractory or intolerant to 2nd generation TKI 2) Aged 3-14 years 3) BSA >=0.6 m2 4) ECOG PS score of 0-2.
Exclusion criteria
Exclusion criteria: 1) Symptoms of central nervous system bleeding of Grade 3 or more according to the CTCAE v4.0. 2) Infection that is difficult to control 3) Females who are pregnant, breastfeeding or possibly pregnant. 4) Down syndrome 5) long QT 6) diabetes mellitus 7) hyperlipidemia 8) Total bilirubin: more than 3 times the upper limit of the reference value adjusted for age. 9) liver cirrhosis 10) severe psychiatric disorder 11) a history of ischemic cardiac disease 12) a history of thrombosis 13) Creatinine: more than 3 times the upper limit of the reference value adjusted for age. 14) a history of renal failure 15) a history of congenital or acquired immunodeficiency syndrome. 16) have not recovered (>Gr1 by CTCAE Ver 4.0) from adverse events due to previous agents with the exception of alopecia 17) have had cytotoxic chemotherapy, intrathecal therapy or radiotherapy within 14 days of starting ponatinib with the exception of other TKI, steroid or intrathecal therapy for diagnosis 18) have received imatinib within 5 days of starting ponatinib 19) have received dasatinib within 3 days of starting ponatinib 20) have received nilotinib within 8 days of starting ponatinib 21) have received bosutinib within 10 days of starting ponatinib 22) malabsorption syndrome 23) intestinal disease that effect on the absorption of ponatinib 24) uncontrolled cardiac disease 25) uncontrolled hypertension 26) hematopoietic stem cell transplant < 3 months prior to enrollment or any evidence of on-going GVHD 27) received any other agent causes long QT 28) second malingnancy other than CML or Ph+ALL 29) received surgical procedure within 14 days of starting ponatinib (with the exception of the insertion of CV catheter or bone marrow biopsy) 30) a history of HIV, HBV or HCV infections 31) lipase: more than 1.5 times the upper limit of the reference value. 32) amylase: more than 1.5 times the upper limit of the reference value
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| FDT (Fixed dosed toxicity) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Major hematologic response, cytogenetic response and molecular response after the treatment of ponatinib 2) Temporal change of minute residual lesions (Ig / TCR PCR MRD, FCM MRD, chimeric gene MRD) of ponatinib 3) Relation between BCR-ABL chimeric gene mutation and MRD disappearance rate / MRD recurrence rate 4) Pharmacokinetics of ponatinib 5) Relationship between ponatinib blood concentration and MRD elimination rate , MRD recurrence rate 6) Accomplishment rate of ponatinib treatment 7) Adverse Event Incidence | — |
Contacts
Public ContactYuichi Kodama
Kagoshima University Hospital
Outcome results
None listed