NCT01746836

Ponatinib Hydrochloride as Second Line Therapy in Treating Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib Mesylate, Dasatinib, or Nilotinib

Ponatinib as Second Line Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase Resistant or Intolerant to Imatinib, Dasatinib or Nilotinib

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well ponatinib hydrochloride works as second line therapy in treating patients with chronic myeloid leukemia in chronic phase that has not responded to initial treatment (first line) with imatinib mesylate, dasatinib, or nilotinib or cannot tolerate imatinib mesylate, dasatinib, or nilotinib. Ponatinib hydrochloride may stop or control the growth of cancer cells by blocking a protein needed for cell growth.

Detailed description

Primary Objectives: * To estimate the proportion of participants with tyrosine kinase inhibitor (TKI)-resistant, chronic phase CML (CP-CML) attaining major cytogenetic response (MCyR) at 6 months of treatment with second line ponatinib therapy. * To estimate the time to toxicity related to ponatinib for patients with TKI-resistant CP-CML. Secondary Objectives: * To estimate the proportion of participants achieving a MCyR, complete cytogenetic response (CCyR), major molecular response (MMR) and complete molecular response (CMR) at 3, 6, 12, 18 and 24 months of treatment with ponatinib after one second generation TKI failure (by resistance). * To estimate the time to CCyR, MMR, MCyR and CMR for participants treated with ponatinib as second line therapy for CP-CML * To evaluate the durations of hematologic, cytogenetic and molecular response to ponatinib after one second generation TKI failure. * To define the time to progression and overall survival for participants with CML in chronic phase treated with ponatinib after one second generation TKI failure. * To evaluate the toxicity profile of ponatinib in participants with CML in chronic phase after one second generation TKI failure. * To evaluate the probability of developing ABL mutations for participants with CML in chronic phase treated with ponatinib after one second generation TKI failure. * To analyze differences in response rates and in prognosis according to pre-treatment mutations and patient characteristics. * To investigate mechanisms of resistance in patients who develop resistance to ponatinib used as second line therapy for CP-CML. * To evaluate symptom burden in participants with CP-CML receiving ponatinib. Exploratory Objectives: • To investigate the presence of miRNA that may be predictive of outcome

Conditions

• Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
• Philadelphia Chromosome Positive, BCR-ABL1 Positive Chronic Myelogenous Leukemia
• Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Blood draws.
DRUG	Ponatinib Hydrochloride	Starting dose: 30 mg by mouth once a day.
OTHER	Quality-of-Life Assessment	Surveys completed.

Timeline

• Start date: 2013-01-17
• Primary completion: 2030-12-31
• Completion: 2030-12-31
• First posted: 2012-12-11
• Last updated: 2026-03-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study