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Tyrosine Kinase Inhibitor

Ponatinib for Chronic Myeloid Leukemia

Phase 2
Recruiting
Led By Jorge Cortes
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Eastern Cooperative Oncology Group (ECOG) performance of 0-2
Diagnosis of Philadelphia chromosome (Ph)-positive or breakpoint cluster region (BCR)-ABL-positive CML in chronic phase
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Study Summary

This trial tests ponatinib hydrochloride as second line therapy for CML patients who didn't respond to initial treatment with imatinib mesylate, dasatinib, or nilotinib, or can't tolerate those treatments. Ponatinib hydrochloride may work by blocking a protein needed for cell growth.

Who is the study for?
This trial is for patients with chronic myeloid leukemia in the chronic phase who didn't respond to or can't tolerate initial treatments like imatinib mesylate, dasatinib, or nilotinib. Participants must have normal organ function tests, be able to consent, and not be pregnant or breastfeeding. They should also have no severe heart disease, uncontrolled high blood pressure, psychiatric disorders, history of certain cardiovascular events, pancreatitis or more than one prior treatment with FDA-approved TKIs.Check my eligibility
What is being tested?
The trial is testing ponatinib hydrochloride as a second line therapy for chronic myeloid leukemia that's resistant or intolerant to first-line drugs. It includes laboratory biomarker analysis and quality-of-life assessments to see if this drug can control cancer cell growth by blocking proteins needed for their proliferation.See study design
What are the potential side effects?
Potential side effects of ponatinib hydrochloride may include risks such as pancreatitis (inflammation of the pancreas), changes in blood pressure and heart rhythm disturbances including prolonged QT interval which affects heart electrical activity.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am able to care for myself and perform daily activities.
Select...
My leukemia is in the chronic phase and tests positive for specific genetic markers.
Select...
I have tried one FDA-approved TKI treatment without success.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
MCyR at 6 months (MCyR6)
Time-to-toxicity defined as any grade 3 or 4 drug-related adverse event that is not responsive to standard therapeutic management and requires permanent treatment discontinuation
Secondary outcome measures
Duration of MCyR
MMR
Time to transformation to accelerated phase CML
+1 more

Side effects data

From 2019 Phase 2 trial • 12 Patients • NCT02265341
75%
Fatigue
58%
Abdominal pain
58%
Constipation
58%
Rash maculo-papular
50%
Nausea
42%
Edema limbs
42%
Platelet count decreased
33%
Alkaline phosphatase increased
25%
Diarrhea
25%
Hypertension
17%
Anorexia
17%
Cough
17%
Vomiting
17%
Alanine aminotransferase increased
8%
Pleural effusion
8%
Anemia
8%
Arthralgia
8%
Back pain
8%
Headache
8%
White blood cell decreased
8%
Hyperglycemia
8%
Hoarseness
8%
Pneumonitis
8%
Dry skin
8%
Sepsis
8%
Fever
8%
Blood bilirubin increased
8%
CD4 lymphocytes decreased
8%
Lymphocyte count decreased
8%
Neutrophil count decreased
8%
Pancreatitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Treatment (Ponatinib Hydrochloride)

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Ponatinib hydrochlorideExperimental Treatment3 Interventions
Patients receive ponatinib hydrochloride PO QD. Treatment continues for up to 5 years in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ponatinib Hydrochloride
2014
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor
2,946 Previous Clinical Trials
1,801,773 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,587 Previous Clinical Trials
41,242,452 Total Patients Enrolled
Jorge CortesPrincipal InvestigatorM.D. Anderson Cancer Center
8 Previous Clinical Trials
348 Total Patients Enrolled

Media Library

Ponatinib Hydrochloride (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT01746836 — Phase 2
Chronic Myelogenous Leukemia Research Study Groups: Ponatinib hydrochloride
Chronic Myelogenous Leukemia Clinical Trial 2023: Ponatinib Hydrochloride Highlights & Side Effects. Trial Name: NCT01746836 — Phase 2
Ponatinib Hydrochloride (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01746836 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Why did researchers design this clinical trial?

"The purpose of this study is to assess the rate of MCyR at 6 months post-treatment (MCyR6) and will secondary measure duration of MCyR, MMR, and time to transformation to accelerated phase CML."

Answered by AI

Are there any other precedents for this type of treatment?

"Ponatinib Hydrochloride is being trialed in 29 active studies, 3 of which are Phase 3 clinical trials. The highest concentration of these research centres for this medication are based in Roma and Gyeongsangbuk-do; however, there are 796 total locations running tests for Ponatinib Hydrochloride."

Answered by AI

What indications does Ponatinib Hydrochloride usually treat?

"Ponatinib Hydrochloride is an effective treatment against acute lymphoblastic leukemia (all), myeloid leukemia, chronic, chronic phase, and leukemia, myeloid, accelerated phase."

Answered by AI

How many people have been selected to participate in this clinical research?

"That is correct. Based on the information available on clinicaltrials.gov, it seems that this study is still open to recruiting patients. The trial was first posted on 1/17/2013 and edited for the last time on 6/15/2022. They are looking for 50 patients from a single site."

Answered by AI

Are we still enrolling individuals in this experiment?

"That is correct. The clinical trial is currently underway and recruiting 50 patients from a single site, as stated on the website clinicaltrials.gov. This information was originally posted on 1/17/2013 and was last updated on 6/15/2022."

Answered by AI

Has Ponatinib Hydrochloride been cleared for use by the FDA?

"Ponatinib Hydrochloride is considered to be a level 2 medication in terms of safety. While there is some evidence that it is safe, none of the clinical trials thus far have been able to confirm its efficacy."

Answered by AI
~19 spots leftby Dec 2030