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Tyrosine Kinase Inhibitor

Ponatinib for Chronic Myeloid Leukemia (OPTIC Trial)

Phase 2

Waitlist Available

Research Sponsored by Ariad Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Have chronic phase-chronic myelogenous leukemia/chronic myeloid leukemia (CP-CML) and have received at least two prior tyrosine kinase inhibitor (TKI) therapies and have demonstrated resistance to treatment OR have documented history of presence of T315I mutation after receiving any number of prior TKI.

Diagnosis of chronic myeloid leukemia (CML) will be made using standard hematopathologic and cytogenetic criteria; CP-CML will be defined by specific criteria.

Must not have

Have a significant bleeding disorder unrelated to CML.

Have undergone autologous or allogeneic stem cell transplant <60 days prior to receiving the first dose of ponatinib; have any evidence of ongoing graft-versus-host disease (GVHD) or GVHD requiring immunosuppressive therapy.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from first dose date of study treatment up to data cut-off: 31 may 2020 (approximately 5 years)

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the effectiveness of a drug called ponatinib for people with a certain type of leukemia who haven't responded to other treatments.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Ponatinib may cause side effects such as liver problems, pancreatitis, high blood pressure, rash or dry skin. It can also affect the heart's rhythm and increase the risk of blood clots or bleeding.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have chronic myeloid leukemia and my previous treatments haven't worked or I have a specific mutation.

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I have been diagnosed with chronic myeloid leukemia in the chronic phase.

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My tests show I have the Philadelphia chromosome in my cancer cells.

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My previous treatment with a TKI drug did not work.

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My kidney function is within normal ranges.

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My pancreas is functioning normally.

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My liver is working well.

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I am able to care for myself and perform daily activities.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a bleeding disorder not caused by my leukemia.

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

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I haven't had certain cancer treatments or recovered from their side effects recently.

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I have been treated with ponatinib before.

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My high blood pressure is not under control.

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I have had cancer before, but it's not one of the few exceptions.

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I have not had major surgery in the last 2 weeks.

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I am currently on IV antibiotics for an infection.

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I have been diagnosed with HIV.

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I have a serious heart condition that is not under control.

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I am not on medications known to cause heart rhythm problems.

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My diabetes is not well-managed.

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I do not have any stomach or bowel problems that affect how I absorb medicine taken by mouth.

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I have had pancreatitis before.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to data cut-off: 31 may 2020 (approximately 5 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to data cut-off: 31 may 2020 (approximately 5 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to data cut-off: 31 may 2020 (approximately 5 years) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of Participants With Molecular Response (MR2: <=1% Breakpoint Cluster Region-Abelson Transcript Level) as Measured by the International Scale (BCR-ABL1IS) at Month 12

Secondary study objectives

Duration of Major Molecular Response (MMR/MR3)

Duration of Response in Responders

Kaplan-Meier Estimate of Duration of Response (DOR) of <=1% BCR-ABL1 IS (MR2) at Months 12 and 24

+13 more

Side effects data

From 2017 Phase 2 trial • 171 Patients • NCT01935336

67%

Grade 2 hypertension

67%

Grade 3 AE

100%

80%

60%

40%

20%

Study treatment Arm

Ponatinib SISH-/ISH+

Ponatinib SISH-/ISH-

Ponatinib SISH+/ISH+

Ponatinib SISH+/ISH-

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ponatinib

2015

Completed Phase 2

~830

0 / 22Eligibility criteria met