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Tyrosine Kinase Inhibitor

Ponatinib + Chemotherapy for Acute Lymphoblastic Leukemia

Phase 1 & 2

Waitlist Available

Research Sponsored by Takeda

Eligibility Criteria Checklist

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

Must have

HSCT: Participants who have experienced relapse after a HSCT are eligible, provided they have no evidence of acute or chronic graft-versus-host disease (GVHD), are not receiving GVHD prophylaxis or treatment, and are at least 90 days posttransplant at the time of enrollment.

a) Involvement of BM with ALL, including one of the following: i. M2 BM (5%-24% lymphoblasts): by morphology with confirmatory testing consisting of at least one of the following: flow cytometry lymphoblasts ≥5%, or BCR-ABL1 fluorescence in situ hybridization, or ≥10-2 leukemic clone identified by immunoglobulin heavy chain-T-cell receptor polymerase chain reaction, OR ii. M3 BM (≥25% lymphoblasts): by morphology, OR iii. Participants with combined BM (as defined above) and extramedullary disease.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 36 months

Awards & highlights

Study Summary

This trial is testing an anticancer drug called ponatinib, given with chemotherapy, to children, teenagers, and young adults up to 21 years of age with leukemia that has relapsed or is resistant to other treatment.

Who is the study for?

This trial is for children, teenagers, and young adults up to age 21 with Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia who have not responded well to other treatments or have a specific mutation (BCR-ABL1 T315I). They should be able to perform daily activities at least half of the time (Karnofsky/Lansky score ≥50%) and recovered from previous treatment side effects. Those with another cancer, uncontrolled health conditions like hypertension or seizures, recent use of certain drugs affecting liver enzymes, or history of severe blood disorders cannot join.Check my eligibility

What is being tested?

The study tests ponatinib tablets/capsules combined with chemotherapy in participants who either can't swallow pills or need smaller doses. The goal is to find the highest safe dose that might help achieve leukemia remission. Participants will take this combination for 10 weeks alongside standard chemotherapy blocks and will be monitored for at least three years.See study design

What are the potential side effects?

Ponatinib may cause cardiovascular issues like high blood pressure or heart problems; it could also affect liver function and increase the risk of bleeding events. Other possible side effects include fatigue, digestive disturbances, skin reactions, headaches, joint pain and an increased risk of infections.

Eligibility Criteria

Inclusion Criteria

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

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I had a stem cell transplant, no GVHD, not on GVHD treatment, and it's been 90 days since.

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My leukemia involves bone marrow with specific levels of lymphoblasts or extramedullary disease.

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I stopped my last cancer treatment due to severe side effects.

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I have been diagnosed with a specific type of leukemia linked to the Philadelphia chromosome.

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My leukemia is positive for a specific genetic change.

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I have recovered from side effects of my previous treatment, except for hair loss.

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My previous cancer treatment, which targeted a specific protein, did not work or caused side effects I couldn't tolerate.

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I am mostly capable of self-care and daily activities.

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My leukemia has specific genetic changes.

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My leukemia has spread to my bone marrow and possibly other areas.

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I am mostly able to carry out daily activities.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 36 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 36 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 36 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy

Phase 2: Complete Remission (CR) Rate at the end of Reinduction Block

Secondary outcome measures

Phase 1 and 2: Number of Participants with Adverse Events (AEs), Serious Adverse Events (SAEs), Venous Thrombotic/Embolic Events (VTEs), and Adverse Events of Special Interest (AESIs)

Phase 1: AUClast: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration for Ponatinib

Phase 1: CR Rate at the end of Reinduction Block

+10 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: PonatinibExperimental Treatment3 Interventions

Ponatinib tablet or age appropriate formulation (AAF) [i.e., capsules with ponatinib minitablets], based on age and weight, in combination with chemotherapy backbone, orally, once daily in both reinduction block and consolidation block (35 days each including 29 days of treatment followed by rest period from chemotherapy for a minimum of 6 days consisting of daily ponatinib only) in Phase 1 to determine RP2D. In Phase 2 participants will receive ponatinib at RP2D in combination with chemotherapy backbone.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ponatinib

2015

Completed Phase 2

~820

0 / 11Eligibility criteria met

Find a Location

Who is running the clinical trial?

TakedaLead Sponsor

1,202 Previous Clinical Trials

4,178,194 Total Patients Enrolled

Study DirectorStudy DirectorTakeda

1,207 Previous Clinical Trials

489,612 Total Patients Enrolled

Study Director Clinical ScienceStudy DirectorTakeda

5 Previous Clinical Trials

1,399 Total Patients Enrolled

Media Library

Ponatinib (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04501614 — Phase 1 & 2

Acute Lymphoblastic Leukemia Research Study Groups: Ponatinib

Acute Lymphoblastic Leukemia Clinical Trial 2023: Ponatinib Highlights & Side Effects. Trial Name: NCT04501614 — Phase 1 & 2

Ponatinib (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04501614 — Phase 1 & 2

Frequently Asked Questions

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

In how many different medical clinics is this research being conducted today?

"This study is available at 16 hospitals, some of which include Children's Mercy Hospital and Clinica in Kansas City, Dana Farber Cancer Institute in Boston, and Children's Hospital of Philadelphia in Philadelphia."

Answered by AI

Can you tell me about other scientific research that has looked at Chemotherapy Agents?

"Right now, there are twenty-nine Chemotherapy Agent trials ongoing with three of them in Phase 3. The largest number of these clinical trials for Chemotherapy Agents are based in Portland, Oregon; however, there are 796 total locations conducting these sorts of medical studies."

Answered by AI

Does the age limit for this trial stop at 85 years old?

"Children aged 1 year up to young adults 21 years old can qualify for this clinical trial. Out of the 480 studies available for people under 18, this is one option. 1255 studies are available for those over 65."

Answered by AI

Are there any open slots left for this research project?

"The trial, which was posted on February 24th 2021 and updated September 22nd 2022 according to clinicaltrials.gov, is looking for patients at this time."

Answered by AI

What is the most popular use for Chemotherapy Agents?

"Chemotherapy agents are a potential treatment for acute lymphoblastic leukemia (ALL), myeloid leukemia, chronic phase and accelerated phase."

Answered by AI

Am I eligible to participate in this research?

"This trial is looking for 68 participants who have leukemia, lymphocytic, acute, l1 between the ages of 1 and 21. The most important criteria applicants are required to meet are as follows: (i) For non-US sites: participants must have relapsed (post 0 or 1 HSCT) or be resistant or intolerant to at least one prior therapy that contained a BCR-ABL-targeted tyrosine kinase inhibitor (TKI), or for US sites: participants who have relapsed (post 0 or 1 HSCT) or are resistant or intolerant to at least one prior therapy that"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

2021. "A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04501614.

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