Trial · NCT04501614
A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
NCT04501614, Phase 1, Phase 2, Terminated, Takeda; results first posted 2025-01-15
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia. Trials, record NCT04501614. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04501614
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
- NCT number
- NCT04501614
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 11 participants, actual
- Lead sponsor
- Takeda
- Conditions
- Pediatric Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia (Ph+ALL), Ph+ Mixed Phenotype Acute Leukemia (MPAL), Philadelphia Chromosome-Like ALL (Ph-like ALL)
- Interventions
- Ponatinib, Chemotherapy Agents
- First posted
- 2020-08-06
- Last updated
- 2025-10-02
- Other registries' IDs
- eudract:2019-002549-39
- Results first posted
- 2025-01-15
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy | Up to Day 35 in Phase 1 | The RP2D is the maximum tolerated dose (MTD) or less. |
| Primary | Phase 2: Percentage of Participants With Complete Remission (CR) at the End of the Reinduction Block | Up to Day 35 in Phase 2 | CR was defined as no circulating blasts and less than (\<)5 percent (%) blasts in the bone marrow (BM); normal maturation of all cellular components in the bone marrow; no extramedullary disease; absolute neutrophil count (ANC) greater than (\>)1000 cells/microliter (μL) (or \>1.0 × 10\^9 cells/liter \[L\]); Platelets \>100,000/μL (or \>100 × 10\^9 platelets/L). |
| Secondary | Phase 1: Number of Participants With CR at the End of Reinduction Block | Day 35 in Phase 1 | CR was defined as no circulating blasts and \<5% blasts in the BM; normal maturation of all cellular components in the bone marrow; no extramedullary disease; ANC \>1000 cells/μL (or \>1.0 × 10\^9 cells/L); Platelets \>100,000/μL (or \>100 × 10\^9 platelets/L). |
| Secondary | Phase 2: Percentage of Ph+ ALL Participants Who Achieved CR at the End of Consolidation Block | Day 70 in Phase 2 | CR was defined as no circulating blasts and \<5% blasts in the bone marrow; normal maturation of all cellular components in the bone marrow; no extramedullary disease; ANC \>1000 cells/μL (or \>1.0 × 10\^9 cells/L); Platelets \>100,000 platelets/μL (or \>100 × 10\^9 platelets/L). |
| Secondary | Phase 2: Percentage of Ph+ ALL Participants With Negative Minimal Residual Disease (MRD) Among Those Who Achieved CR | Up to Day 70 (end of consolidation block) in Phase 2 | MRD negative rate is the percentage of participants who achieve MRD negative status by evaluation of bone marrow aspirate at \<0.01% threshold. |
| Secondary | Phase 2: Percentage of Participants Who Relapsed or Progressed Following Reinduction and Consolidation | Up to 3 years of follow-up in Phase 2 | |
| Secondary | Phase 2: Event-free Survival (EFS) | Up to 3 years of follow-up in Phase 2 | EFS was defined as time from date of enrollment until death due to any cause; refractory to treatment (defined as failure to achieve CR by end of the consolidation block) or relapse from CR. CR was defined as no circulating blasts and \<5% blasts in the bone marrow; normal maturation of all cellular components in the bone marrow; no extramedullary disease; ANC \>1000 cells/μL (or \>1.0 × 10\^9 cells/L); Platelets \>100,000 platelets/μL (or \>100 × 10\^9 platelets/L). |
| Secondary | Phase 2: Progression-free Survival (PFS) | Up to 3 years of follow-up in Phase 2 | PFS was defined as time from date of enrolment until death related to disease under study; disease progression (clinical deterioration associated with disease process, including evidence of increasing blasts in the bone marrow from baseline and/or evidence of new organ involvement) or relapse from CR. CR was defined as no circulating blasts and \<5% blasts in the bone marrow; normal maturation of all cellular components in the bone marrow; no extramedullary disease; ANC \>1000 cells/μL (or \>1.0 × 10\^9 cells/L); Platelets \>100,000 platelets/μL (or \>100 × 10\^9 platelets/L). |
| Secondary | Phase 2: Overall Survival (OS) | Up to 3 years of follow-up in Phase 2 | OS was defined as time from first dose of ponatinib until death due to any cause. |
| Secondary | Phase 2: Duration of Response (DOR) | Up to 3 years of follow-up in Phase 2 | DOR was defined as the interval between the first assessment at which the criteria for CR are met until the time at which relapse from CR occurs. CR was defined as no circulating blasts and \<5% blasts in the bone marrow; normal maturation of all cellular components in the bone marrow; no extramedullary disease; ANC \>1000 cells/μL (or \>1.0 × 10\^9 cells/L); Platelets \>100,000 platelets/μL (or \>100 × 10\^9 platelets /L). |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy | Up to Day 35 in Phase 1 | The Safety Population included all participants who received at least 1 dose of ponatinib. | milligrams per square meter (mg/m^2) | NUMBER |
| PRIMARY | Phase 2: Percentage of Participants With Complete Remission (CR) at the End of the Reinduction Block | Up to Day 35 in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 1: Number of Participants With CR at the End of Reinduction Block | Day 35 in Phase 1 | The Safety Population included all participants who received at least 1 dose of ponatinib. Overall number of participants analyzed is the number of participants with data available for analysis. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Phase 2: Percentage of Ph+ ALL Participants Who Achieved CR at the End of Consolidation Block | Day 70 in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 2: Percentage of Ph+ ALL Participants With Negative Minimal Residual Disease (MRD) Among Those Who Achieved CR | Up to Day 70 (end of consolidation block) in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 2: Percentage of Participants Who Relapsed or Progressed Following Reinduction and Consolidation | Up to 3 years of follow-up in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 2: Event-free Survival (EFS) | Up to 3 years of follow-up in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 2: Progression-free Survival (PFS) | Up to 3 years of follow-up in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 2: Overall Survival (OS) | Up to 3 years of follow-up in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. | ||
| SECONDARY | Phase 2: Duration of Response (DOR) | Up to 3 years of follow-up in Phase 2 | The study enrollment was terminated per protocol due to DLTs observed in both dose cohorts in Phase 1 and the RP2D could not be determined leading to study termination by the Sponsor before Phase 2 could be initiated. No data was collected as no participants were enrolled in Phase 2. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy | Ponatinib 30 mg Adult-Equivalent | NA | milligrams per square meter (mg/m^2) | ||
| Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy | Ponatinib 15 mg Adult-Equivalent | NA | milligrams per square meter (mg/m^2) | ||
| Phase 1: Number of Participants With CR at the End of Reinduction Block | Ponatinib 30 mg Adult-Equivalent | 1 | Participants | ||
| Phase 1: Number of Participants With CR at the End of Reinduction Block | Ponatinib 15 mg Adult-Equivalent | 2 | Participants | ||
| Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib | Ponatinib 30 mg Adult-Equivalent | Day 1 | 65.8 | Geometric Coefficient of Variation 29.5 | nanograms/milliliter (ng/mL) |
| Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib | Ponatinib 15 mg Adult-Equivalent | Day 1 | 24.9 | Geometric Coefficient of Variation 71.1 | nanograms/milliliter (ng/mL) |
| Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib | Ponatinib 30 mg Adult-Equivalent | Day 22 | 183 | Geometric Coefficient of Variation NA | nanograms/milliliter (ng/mL) |
| Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib | Ponatinib 15 mg Adult-Equivalent | Day 22 | 47.7 | Geometric Coefficient of Variation 92.8 | nanograms/milliliter (ng/mL) |
| Phase 1: Tmax: Time of First Occurrence of Cmax for Ponatinib | Ponatinib 30 mg Adult-Equivalent | Day 1 | 6.00 | Full Range 2.0 to 7.5 | hours |
| Phase 1: Tmax: Time of First Occurrence of Cmax for Ponatinib | Ponatinib 15 mg Adult-Equivalent | Day 1 | 3.99 | Full Range 3.8 to 4.1 | hours |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ponatinib 30 mg Adult-Equivalent | 7 | |
| Overall Study | STARTED | Ponatinib 15 mg Adult-Equivalent | 4 | |
| Overall Study | COMPLETED | Ponatinib 30 mg Adult-Equivalent | 0 | |
| Overall Study | COMPLETED | Ponatinib 15 mg Adult-Equivalent | 0 | |
| Overall Study | NOT COMPLETED | Ponatinib 30 mg Adult-Equivalent | 7 | |
| Overall Study | NOT COMPLETED | Ponatinib 15 mg Adult-Equivalent | 4 |
Source ClinicalTrials.gov
Adverse events342
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal distension | Gastrointestinal disorders | Ponatinib 30 mg Adult-Equivalent | 1 | 7 | |
| other | Abdominal distension | Gastrointestinal disorders | Ponatinib 15 mg Adult-Equivalent | 0 | 4 | |
| other | Abdominal pain | Gastrointestinal disorders | Ponatinib 30 mg Adult-Equivalent | 2 | 7 | |
| other | Abdominal pain | Gastrointestinal disorders | Ponatinib 15 mg Adult-Equivalent | 2 | 4 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ponatinib 30 mg Adult-Equivalent | 1 | 7 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Ponatinib 15 mg Adult-Equivalent | 0 | 4 | |
| other | Activated partial thromboplastin time prolonged | Investigations | Ponatinib 30 mg Adult-Equivalent | 2 | 7 | |
| other | Activated partial thromboplastin time prolonged | Investigations | Ponatinib 15 mg Adult-Equivalent | 1 | 4 | |
| other | Agitation | Psychiatric disorders | Ponatinib 30 mg Adult-Equivalent | 1 | 7 | |
| other | Agitation | Psychiatric disorders | Ponatinib 15 mg Adult-Equivalent | 0 | 4 |
1 to 10 of 342Source ClinicalTrials.gov
See all 342 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ponatinib 30 mg Adult-Equivalent | 0 | 7 |
| Total, all-cause mortality | Ponatinib 15 mg Adult-Equivalent | 0 | 4 |
| Total, other adverse events | Ponatinib 30 mg Adult-Equivalent | 7 | 7 |
| Total, other adverse events | Ponatinib 15 mg Adult-Equivalent | 4 | 4 |
| Total, serious adverse events | Ponatinib 30 mg Adult-Equivalent | 5 | 7 |
| Total, serious adverse events | Ponatinib 15 mg Adult-Equivalent | 2 | 4 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Ponatinib 30 mg Adult-Equivalent vs Ponatinib 15 mg Adult-Equivalent: Phase 1: Number of Participants With CR at the End of Reinduction Block | Trial comparisons |
| Ponatinib 30 mg Adult-Equivalent vs Ponatinib 15 mg Adult-Equivalent: deaths from any cause | Trial comparisons |
| Ponatinib 30 mg Adult-Equivalent vs Ponatinib 15 mg Adult-Equivalent: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04501614
GET https://conductscience.org/api/trials/v1/NCT04501614/results?page=2