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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 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

Find a Location

Who is running the clinical trial?

TakedaLead Sponsor
1,208 Previous Clinical Trials
4,187,849 Total Patients Enrolled
Study DirectorStudy DirectorTakeda
1,221 Previous Clinical Trials
499,906 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
~9 spots leftby Dec 2024
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