Ponatinib for Chronic Myeloid Leukemia
(OPTIC Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests ponatinib, a medication, to evaluate its effectiveness for people with chronic myeloid leukemia (CML) who have not responded to other treatments or have a specific genetic mutation. The study examines three different doses of ponatinib to identify the most effective option. It seeks participants who have tried at least two other therapies without success or have the T315I mutation in their leukemia. As a Phase 2 trial, this research measures ponatinib's effectiveness in an initial, smaller group, offering participants a chance to potentially benefit from a promising treatment.
Do I need to stop my current medications for the trial?
The trial requires that you stop taking any approved tyrosine kinase inhibitors (TKIs) or investigational agents at least 2 weeks before starting the study drug. Other medications like interferon, cytarabine, or immunotherapy must be stopped 14 days prior, and any cytotoxic chemotherapy or radiotherapy must be stopped 28 days before the trial.
Is there any evidence suggesting that ponatinib is likely to be safe for humans?
Research has shown that ponatinib is generally effective, though some safety concerns exist. In past studies, serious side effects were reported. For instance, one study found that 9% of patients experienced life-threatening side effects, such as blood vessel issues, infections, and severe bleeding.
Other studies have identified blood-related side effects, including low platelet levels (cells that help blood clot), low white blood cell counts (which fight infections), and anemia (low red blood cells). Despite these risks, real-world data from Belgium suggested that ponatinib is relatively safe for treating certain types of leukemia. This indicates that while risks are present, many patients tolerate the treatment well.
Ponatinib has already received approval for certain conditions, indicating that its safety is well-studied. However, it is important to weigh both the benefits and risks before joining a trial. Always consult a healthcare provider to determine if this treatment is suitable.12345Why are researchers excited about this study treatment for chronic myeloid leukemia?
Researchers are excited about ponatinib for chronic myeloid leukemia (CML) because it targets the BCR-ABL1 fusion protein, which is a key driver of this cancer. Unlike standard treatments like imatinib, dasatinib, and nilotinib, ponatinib is effective against mutations that make CML resistant to these drugs, especially the T315I mutation. Ponatinib's ability to inhibit a broader range of mutations offers hope for patients who have exhausted other options, providing a critical alternative in the fight against CML.
What evidence suggests that ponatinib could be an effective treatment for chronic myeloid leukemia?
Research has shown that ponatinib effectively treats chronic myeloid leukemia (CML), particularly in patients unresponsive to other treatments. One study found that ponatinib led to strong and lasting improvements in patients who had tried other drugs before. In this trial, participants will receive different doses of ponatinib: Cohort A will receive 45 mg, Cohort B will receive 30 mg, and Cohort C will receive 15 mg. Previous studies have shown good results with varying doses, with many patients experiencing a major reduction in cancer markers in their blood. These findings support ponatinib as a promising option for people with CML who have limited treatment choices.678910
Who Is on the Research Team?
Study Director Clinical Science
Principal Investigator
Takeda
Are You a Good Fit for This Trial?
This trial is for adults with chronic phase chronic myeloid leukemia (CP-CML) who have tried at least two tyrosine kinase inhibitors without success or have a specific mutation (T315I). Participants must be in good health otherwise, with proper liver, kidney, and pancreatic function, no major heart issues, and not pregnant. They should also agree to use effective contraception.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Main Treatment
Participants receive ponatinib at one of three starting doses (45 mg, 30 mg, or 15 mg) daily in 28-day cycles until specific response criteria or up to 24 cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
Optional Treatment Continuation
Participants may enter an optional treatment continuation period following completion of the main treatment period or early withdrawal
What Are the Treatments Tested in This Trial?
Interventions
- Ponatinib
Ponatinib is already approved in United States, European Union, Canada for the following indications:
- Chronic myeloid leukemia (CML)
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
- Acute lymphoblastic leukemia (ALL)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ariad Pharmaceuticals
Lead Sponsor
Takeda
Lead Sponsor
Dr. Naoyoshi Hirota
Takeda
Chief Medical Officer since 2020
MD from University of Tokyo
Christophe Weber
Takeda
Chief Executive Officer since 2015
PhD in Molecular Biology from Université de Montpellier