Trial Outcomes & Findings for A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (NCT NCT04501614)

NCT ID: NCT04501614

Last Updated: 2025-10-02

Results Overview

The RP2D is the maximum tolerated dose (MTD) or less.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 11 participants
• Primary outcome timeframe: Up to Day 35 in Phase 1
• Results posted on: 2025-10-02

Participant Flow

Participants with a diagnosis of Philadelphia Chromosome-Positive (Ph+) Acute Lymphoblastic Leukemia (ALL) took part in the study at various investigative sites globally from 24 February 2021 to 19 July 2024.

Participants were enrolled in Phase 1 and received ponatinib per protocol specifications (presented per dose regimen here). Due to the observation of multiple dose-limiting toxicities (DLTs) in Phase 1, study enrollment was terminated per protocol and no recommended phase 2 dose (RP2D) could be determined. The Sponsor terminated the study following a 6-month follow-up and no participants were enrolled in Phase 2 of the study, thus, no Phase 2 results are presented here as it was not conducted.

Participant milestones

Measure	Ponatinib 30 mg Adult-Equivalent Participants received weight-based dose of ponatinib tablets 30 milligrams (mg) adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Overall Study STARTED	7	4
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	7	4

Reasons for withdrawal

Measure	Ponatinib 30 mg Adult-Equivalent Participants received weight-based dose of ponatinib tablets 30 milligrams (mg) adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Overall Study Study Terminated by Sponsor	6	3
Overall Study Withdrawal by Participant (Parent/Legal Guardian)	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Ponatinib 30 mg Adult-Equivalent n=7 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Total n=11 Participants Total of all reporting groups
Age, Continuous	12.1 years STANDARD_DEVIATION 2.41 • n=5 Participants	13.5 years STANDARD_DEVIATION 3.70 • n=7 Participants	12.6 years STANDARD_DEVIATION 2.84 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to Day 35 in Phase 1

Population: The Safety Population included all participants who received at least 1 dose of ponatinib.

The RP2D is the maximum tolerated dose (MTD) or less.

Outcome measures

Measure	Ponatinib 30 mg Adult-Equivalent n=7 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Phase 1: Recommended Phase 2 Dose (RP2D) of Ponatinib in Combination With Chemotherapy	NA milligrams per square meter (mg/m^2) Due to DLTs observed in both dose cohorts, study enrollment was terminated per protocol and participants were discontinued from study treatment per independent data monitoring committee (IDMC) advice followed by study termination by Sponsor. Thus, the RP2D could not be determined.	NA milligrams per square meter (mg/m^2) Due to DLTs observed in both dose cohorts, study enrollment was terminated per protocol and participants were discontinued from study treatment per independent data monitoring committee (IDMC) advice followed by study termination by Sponsor. Thus, the RP2D could not be determined.

PRIMARY outcome

Timeframe: Up to Day 35 in Phase 2

Population: 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.

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).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 35 in Phase 1

Population: 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.

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).

Outcome measures

Measure	Ponatinib 30 mg Adult-Equivalent n=6 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=3 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Phase 1: Number of Participants With CR at the End of Reinduction Block	1 Participants	2 Participants

SECONDARY outcome

Timeframe: Day 70 in Phase 2

Population: 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.

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).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to Day 70 (end of consolidation block) in Phase 2

Population: 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.

MRD negative rate is the percentage of participants who achieve MRD negative status by evaluation of bone marrow aspirate at <0.01% threshold.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 3 years of follow-up in Phase 2

Population: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 3 years of follow-up in Phase 2

Population: 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.

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).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 3 years of follow-up in Phase 2

Population: 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.

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).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 3 years of follow-up in Phase 2

Population: 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.

OS was defined as time from first dose of ponatinib until death due to any cause.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 3 years of follow-up in Phase 2

Population: 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.

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).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 3 years of follow-up in Phase 2

Population: 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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Predose and at multiple timepoints post-dose up to 24 hours on Days 1 and 22 in Phase 1

Population: The Pharmacokinetic (PK) Analysis Population included all participants in the Phase 1 portion of the study who had sufficient ponatinib dosing data and concentration-time data to permit the calculation of ponatinib PK parameters. Number analyzed is the number of participants with data available for analysis for the specified timepoint.

Outcome measures

Measure	Ponatinib 30 mg Adult-Equivalent n=7 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib Day 1	65.8 nanograms/milliliter (ng/mL) Geometric Coefficient of Variation 29.5	24.9 nanograms/milliliter (ng/mL) Geometric Coefficient of Variation 71.1
Phase 1: Cmax: Maximum Observed Plasma Concentration for Ponatinib Day 22	183 nanograms/milliliter (ng/mL) Geometric Coefficient of Variation NA Geometric coefficient of variation (CV) was not estimable for a single participant.	47.7 nanograms/milliliter (ng/mL) Geometric Coefficient of Variation 92.8

SECONDARY outcome

Timeframe: Predose and at multiple timepoints post-dose up to 24 hours on Days 1 and 22 in Phase 1

Population: The PK Analysis Population included all participants in the Phase 1 portion of the study who had sufficient ponatinib dosing data and concentration-time data to permit the calculation of ponatinib PK parameters. Number analyzed is the number of participants with data available for analysis for the specified timepoint.

Outcome measures

Measure	Ponatinib 30 mg Adult-Equivalent n=7 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Phase 1: Tmax: Time of First Occurrence of Cmax for Ponatinib Day 1	6.00 hours Interval 2.0 to 7.5	3.99 hours Interval 3.8 to 4.1
Phase 1: Tmax: Time of First Occurrence of Cmax for Ponatinib Day 22	4.08 hours Full range was not estimable for a single participant.	3.92 hours Interval 2.0 to 5.8

SECONDARY outcome

Timeframe: Predose and at multiple timepoints post-dose up to 24 hours on Days 1 and 22 in Phase 1

Population: The PK Analysis Population included all participants in the Phase 1 portion of the study who had sufficient ponatinib dosing data and concentration-time data to permit the calculation of ponatinib PK parameters. Number analyzed is the number of participants with data available for analysis for the specified timepoint.

Outcome measures

Measure	Ponatinib 30 mg Adult-Equivalent n=7 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Phase 1: AUClast: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration for Ponatinib Day 1	883 hours*nanograms/milliliter (h*ng/mL) Geometric Coefficient of Variation 22.8	264 hours*nanograms/milliliter (h*ng/mL) Geometric Coefficient of Variation 62.1
Phase 1: AUClast: Area Under the Plasma Concentration-Time Curve From Time 0 to the Time of the Last Quantifiable Concentration for Ponatinib Day 22	2800 hours*nanograms/milliliter (h*ng/mL) Geometric Coefficient of Variation NA Geometric CV was not estimable for a single participant.	815 hours*nanograms/milliliter (h*ng/mL) Geometric Coefficient of Variation 83.2

SECONDARY outcome

Timeframe: Up to 34.8 months in Phase 1

Population: The Safety Population included all participants who received at least 1 dose of ponatinib.

An adverse event (AE) is defined as any untoward medical occurrence in clinical study participant, temporally associated with use of study intervention, whether or not occurrence was considered related to the study intervention including any unfavorable & unintended sign (e.g., clinically significant abnormal laboratory finding), symptom/disease. TEAE: any AE that occurs after administration of first dose of any study drug & through 30 days after last dose of any study drug. Serious TEAE:AE that at any dose resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent/significant disability or incapacity, was congenital anomaly/birth defect, was medically important event. AESI: Protocol-defined AEs resulting in compromise of organ function or other significant consequences. VTEs were identified as AESIs for ponatinib and included arterial, VTEs that meet criteria for serious TEAEs.

Outcome measures

Measure	Ponatinib 30 mg Adult-Equivalent n=7 Participants Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 Participants Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Phase 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, Venous Thrombotic/Embolic Events (VTEs), and Adverse Events of Special Interest (AESIs) Serious TEAEs	5 Participants	2 Participants
Phase 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, Venous Thrombotic/Embolic Events (VTEs), and Adverse Events of Special Interest (AESIs) VTEs	0 Participants	0 Participants
Phase 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, Venous Thrombotic/Embolic Events (VTEs), and Adverse Events of Special Interest (AESIs) TEAEs	7 Participants	4 Participants
Phase 1: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs, Venous Thrombotic/Embolic Events (VTEs), and Adverse Events of Special Interest (AESIs) AESIs	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 30 days after last dose of ponatinib in Phase 2

Population: 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.

An AE is defined as any untoward medical occurrence in clinical study participant, temporally associated with use of study intervention, whether or not occurrence was considered related to the study intervention including any unfavorable & unintended sign (e.g., clinically significant abnormal laboratory finding), symptom/disease. TEAE: any AE that occurs after administration of first dose of any study drug & through 30 days after last dose of any study drug. Serious TEAE:AE that at any dose resulted in death, was life-threatening, required inpatient hospitalization/prolongation of existing hospitalization, resulted in persistent/significant disability or incapacity, was congenital anomaly/birth defect, was medically important event. AESI: Protocol-defined AEs resulting in compromise of organ function or other significant consequences. VTEs were identified as AESIs for ponatinib and included arterial, VTEs that meet criteria for serious TEAEs.

Outcome measures

Outcome data not reported

Adverse Events

Ponatinib 30 mg Adult-Equivalent

Serious events: 5 serious events

Other events: 7 other events

Deaths: 0 deaths

Ponatinib 15 mg Adult-Equivalent

Serious events: 2 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Measure	Ponatinib 30 mg Adult-Equivalent n=7 participants at risk Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 participants at risk Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Investigations Alanine aminotransferase increased	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Bilirubin conjugated increased	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Catheter site haemorrhage	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Cerebral haemorrhage	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Cerebral ventricle dilatation	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Disseminated mucormycosis	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Hepatobiliary disorders Hepatic function abnormal	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Hepatobiliary disorders Hepatotoxicity	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypertriglyceridaemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Influenza	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Pneumonia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Seizure	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.

Other adverse events

Measure	Ponatinib 30 mg Adult-Equivalent n=7 participants at risk Participants received weight-based dose of ponatinib tablets 30 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.	Ponatinib 15 mg Adult-Equivalent n=4 participants at risk Participants received weight-based dose of ponatinib tablets 15 mg adult-equivalent, orally, once daily in combination with chemotherapy backbone per standard regimen at the investigator's discretion in both reinduction block and consolidation block (35 days each including 29 days of treatment in combination with chemotherapy followed by a minimum 6-day rest period from chemotherapy with daily ponatinib only) up to Day 70 (end of consolidation) followed by optional ponatinib continuation in Phase 1.
Gastrointestinal disorders Abdominal distension	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Abdominal pain	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	50.0% 2/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Abdominal pain upper	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Activated partial thromboplastin time prolonged	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Psychiatric disorders Agitation	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Alanine aminotransferase increased	100.0% 7/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	100.0% 4/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Injury, poisoning and procedural complications Allergic transfusion reaction	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Amylase increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Anaemia	100.0% 7/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	100.0% 4/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Anal erythema	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Anal inflammation	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Anal ulcer	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Antithrombin III decreased	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Psychiatric disorders Anxiety	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Aphthous ulcer	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Musculoskeletal and connective tissue disorders Arthralgia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	50.0% 2/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Ascites	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Aspartate aminotransferase increased	100.0% 7/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Asthenia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Cardiac disorders Atrial thrombosis	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Musculoskeletal and connective tissue disorders Back pain	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Bacteraemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Bilirubin conjugated increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	50.0% 2/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood albumin decreased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood bilirubin increased	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood bilirubin unconjugated increased	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood calcium decreased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood creatinine increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood fibrinogen decreased	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood glucose increased	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood lactate dehydrogenase increased	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood magnesium decreased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood phosphorus decreased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Blood triglycerides increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations C-reactive protein increased	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations COVID-19	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Cardiac disorders Cardiac failure	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Cerebral haemorrhage	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Chest discomfort	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Chest pain	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Coagulopathy	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Psychiatric disorders Confusional state	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Conjunctivitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Constipation	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	50.0% 2/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Respiratory, thoracic and mediastinal disorders Cough	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Renal and urinary disorders Cystitis noninfective	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Vascular disorders Deep vein thrombosis	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Dermatitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Disseminated mucormycosis	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Dry skin	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Dysgeusia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Eczema asteatotic	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Electrocardiogram QT prolonged	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Electrocardiogram T wave abnormal	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Eosinophil count decreased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Erythema	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Facial pain	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Faeces hard	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Fatigue	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Flatulence	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Folliculitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Immune system disorders Food allergy	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Hepatobiliary disorders Gallbladder oedema	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Gamma-glutamyltransferase increased	85.7% 6/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Gastrointestinal inflammation	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Gingival erythema	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Glossitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Glycosylated haemoglobin increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Headache	71.4% 5/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Hemiparesis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Hemiplegia	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Hepatobiliary disorders Hepatic steatosis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Herpes simplex	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Hepatobiliary disorders Hyperbilirubinaemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Hypercoagulation	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hyperglycaemia	42.9% 3/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hyperlipidaemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypermagnesaemia	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hyperphosphataemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Vascular disorders Hypertension	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypertriglyceridaemia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	100.0% 4/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hyperuricaemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypoalbuminaemia	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypocalcaemia	42.9% 3/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypochloraemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Hypofibrinogenaemia	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypokalaemia	42.9% 3/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypomagnesaemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hyponatraemia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Metabolism and nutrition disorders Hypophosphataemia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Vascular disorders Hypotension	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Endocrine disorders Hypothyroidism	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Infection	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Psychiatric disorders Insomnia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Leukopenia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Injury, poisoning and procedural complications Ligament sprain	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Injury, poisoning and procedural complications Lip injury	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Lipase increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Lymphocyte count decreased	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	50.0% 2/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Lymphopenia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Malaise	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Mental impairment	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Monocyte count increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Injury, poisoning and procedural complications Mouth injury	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Mouth ulceration	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Musculoskeletal and connective tissue disorders Muscle spasms	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Musculoskeletal and connective tissue disorders Myalgia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Myringitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Nasopharyngitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Nausea	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Neuropathy peripheral	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Neutropenia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Neutrophil count decreased	71.4% 5/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Neutrophil count increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Night sweats	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Non-cardiac chest pain	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Oral disorder	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Oral infection	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Pain	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Musculoskeletal and connective tissue disorders Pain in extremity	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Pain of skin	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Pancreatitis acute	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Papule	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Paraesthesia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Parotitis	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Peripheral motor neuropathy	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Peripheral sensorimotor neuropathy	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Nervous system disorders Peripheral sensory neuropathy	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Platelet count decreased	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Platelet count increased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Respiratory, thoracic and mediastinal disorders Pleural effusion	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Pneumonia	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Protein total decreased	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Protein urine present	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations Prothrombin time prolonged	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Pyrexia	42.9% 3/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Rash	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Rash erythematous	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Rash macular	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Rash papular	28.6% 2/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Respiratory tract infection	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Rhinitis	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Cardiac disorders Sinus bradycardia	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Cardiac disorders Sinus tachycardia	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Skin exfoliation	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin papilloma	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Skin and subcutaneous tissue disorders Skin toxicity	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Stomatitis	42.9% 3/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	50.0% 2/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
General disorders Swelling	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Blood and lymphatic system disorders Thrombocytopenia	42.9% 3/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Tongue ulceration	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Upper respiratory tract infection	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Infections and infestations Urinary tract infection	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Injury, poisoning and procedural complications Vascular injury	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Cardiac disorders Ventricular hypertrophy	0.00% 0/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Gastrointestinal disorders Vomiting	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	25.0% 1/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations White blood cell count decreased	57.1% 4/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	75.0% 3/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.
Investigations White blood cells urine positive	14.3% 1/7 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.	0.00% 0/4 • Up to 34.8 months in Phase 1 The Safety Population included all participants who received at least 1 dose of ponatinib. The study was terminated following the Sponsor's decision attributed to DLTs observed in Phase 1. Hence, no participants were enrolled in Phase 2 of the study and no data for the same could be presented.

Additional Information

Medical Director

Takeda

Phone: +1-877-825-3327

Email: TrialDisclosures@takeda.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place