Ponatinib for Chronic Myeloid Leukemia
(OPTIC Trial)

Recruiting in Palo Alto (17 mi)

+85 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Ariad Pharmaceuticals

No Placebo Group

Prior Safety Data

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?The purpose of this study is to characterize the efficacy of ponatinib administered in 3 starting doses (45 mg, 30 mg, and 15 mg daily) in participants with CP-CML who are resistant to prior tyrosine-kinase inhibitor (TKI) therapy or have T315I mutation, as measured by \<=1 % Breakpoint Cluster Region-Abelson Transcript Level using International Scale (BCR-ABL1IS) at 12 months.

Eligibility Criteria

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

Provide written informed consent.

Have normal QT interval corrected (Frederica) (QTcF) interval on screening electrocardiogram (ECG) evaluation.

Have a negative pregnancy test documented prior to enrollment (for females of childbearing potential).

+11 more

Exclusion Criteria

I have a bleeding disorder not caused by my leukemia.

I had a stem cell transplant less than 60 days ago and am experiencing or being treated for graft-versus-host disease.

Have active CNS disease as evidenced by cytology or pathology.

+18 more

Participant Groups

The study tests three different starting doses of ponatinib (45 mg, 30 mg, and 15 mg daily) to see how well they work in patients resistant to previous treatments or those with the T315I mutation. The goal is to achieve a very low level of cancer cells after one year as measured by a specific lab test.

3Treatment groups

Experimental Treatment

Group I: Cohort C: Ponatinib 15 mgExperimental Treatment1 Intervention

Participants received ponatinib 15 mg orally once daily up to data cut-off: 31 May 2020 in each 28 day Cycle.

Group II: Cohort B: Ponatinib 30 mgExperimental Treatment1 Intervention

Ponatinib 30 mg orally once daily in each 28 day Cycle until achievement of ≤1% BCR-ABL1IS. Once ≤1% BCR-ABL1IS up to data cut-off: 31 May 2020. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily.

Group III: Cohort A: Ponatinib 45 mgExperimental Treatment1 Intervention

Ponatinib 45 mg orally once daily in each 28-day cycle until achievement of ≤1% BCR-ABL1IS up to data cut-off: 31 May 2020. Once ≤1% BCR-ABL1IS was achieved, participants received reduced dose of ponatinib 15 mg orally once daily.

Ponatinib is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Iclusig for: