This phase 2 trial is evaluating the effectiveness of ponatinib (Iclusig) plus chemotherapy for patients with acute lymphoblastic leukemia (ALL) that is positive for the Philadelphia chromosome (Ph+) or the BCR-ABL gene (BCR-ABL+). The main outcome to be measured will be the number of patients who respond to treatment. This study is recruiting in Houston, Texas, US.
ALL is a type of cancer of the blood and bone marrow. It is often associated with abnormal genes, such as Ph+ or BCR-ABL+. Chemotherapy is the typical first treatment for patients with ALL. However, some patients may not be able to tolerate intense chemotherapy. Targeted therapy is another option, either alone or combined with other treatments. Ponatinib is a targeted therapy. It blocks some of the enzymes that cancer cells need to grow.
This study is evaluating the effectiveness of ponatinib plus low-dose chemotherapy for patients with ALL that is Ph+ or BCR-ABL+. The main outcome to be measured will be the number of patients who respond to treatment, including those who have a complete disappearance of all signs of cancer.
Who are they looking for?
This study is recruiting 60 patients. This study is looking for patients aged 60 or older with previously untreated ALL. Patients who have had 1 to 2 courses of chemotherapy only may participate. Patients aged 18 or older with relapsed or unresponsive ALL or chronic myeloid leukemia (CML) are also recruited. All patients must agree to use contraception during treatment and for 4 months after finishing treatment. Patients should have adequate liver and kidney function.
Patients with uncontrolled infections, hepatitis B, pancreas disease, or heart disease cannot participate. Patients with a history of alcohol abuse, uncontrolled high blood pressure, or severe heart failure cannot participate. Patients with a history of bleeding disorders cannot participate. Patients treated with anti-leukemia therapy within 1 week of starting the study cannot participate.
How will it work
All patients will receive low-dose chemotherapy combined with ponatinib. All patients will also receive rituximab (Rituxan) and granulocyte colony stimulating factor (G-CSF) treatment. This is a type of growth factor that helps white blood cells recover after treatment. Patients will also receive low-dose chemotherapy.
The main outcome to be measured will be the number of patients who respond to treatment or have a complete disappearance of all signs of cancer. The number of patients who remain alive without cancer recurrence, growth, or spread will also be measured. Safety will also be measured as the number of patients who have side effects from treatment. Patients will be followed-up at 1 month after treatment for up to 6 years.