Symptoms and General Pathology

All Conditions

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Blood and Lymph Conditions

Immune System Diseases

Rare Diseases

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Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Leukemia, Myeloid

Leukemia, Myeloid, Acute

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Leukemia, Myeloid, Chronic-Phase

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Blast Crisis

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Hematologic Diseases

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Chronic Disease

Disease Attributes

Cell Transformation, Neoplastic

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Myeloid Leukemia

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Acute Graft Versus Host Disease

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Antineoplastic Agents

All Drugs and Chemicals

Amino Acids

Ponatinib

Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Tyrosine

Phase 1: Ponatinib administered according to age-based cohort doses and formulations to determine the maximum tolerated dose and recommended Phase 2 dose. Phase 2: Ponatinib administered at the recommended Phase 2 dose.

Ponatinib administered as a tablet or age-appropriate formulation for pediatric participants according to age-based cohort assignment.

Incyte Corporation Call Center (ex-US)

Mohammed-El-Amine Bensmaine, MD

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to \< 18 years with advanced leukemias, lymphomas, and solid tumors.

Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors

An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors in Pediatric Participants

Defined as the occurrence of any protocol-defined toxicities occurring after dosing and up to and including Day 28, except those toxicities with a clear alternative explanation.

Defined as complete cytogenetic response (CCyR) or partial cytogenetic response (PCyR) as assessed by conventional cytogenetics or fluorescence in situ hybridization (FISH).

Assessed by polymerase chain reaction (PCR).

Assessed by conventional cytogenetics, FISH, or PCR.

According to Lugano criteria based on computed tomography (CT) or magnetic resonance imaging (MRI) (or positron emission tomography \[PET\]).

Defined as the percentage of participants having CR or PR, as determined by investigator assessment of radiographic disease per tumors per RANO for central nervous system (CNS) tumors or Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) for other solid tumors based on CT or MRI (or PET).

Area under the steady-state plasma or serum concentration-time curve from Hour 0 to 24.

Apparent terminal-phase disposition half-life.

Apparent oral dose clearance at steady state.

Apparent oral dose volume of distribution.

Defined as CCyR or PCyR as assessed by conventional cytogenetics or FISH.

Defined as the interval from the date of the first dose of study treatment to first response.

Defined as the interval between the first assessment at which the criteria for response are met until the criteria for progression are met.

Defined as the interval from the date of the first dose of study treatment until the date of progression of disease or the date of death from any cause, whichever is earlier.

Defined as the interval from the date of the first dose of study treatment until death from any cause.

Assessed by conventional cytogenetics, FISH, or q-PCR.

According to Lugano criteria based on CT or MRI (or PET).

Defined as the percentage of participants having CR or PR, as determined by investigator assessment of radiographic disease per tumors per RANO for CNS tumors or RECIST v1.1 for other solid tumors based on CT or MRI (or PET).

Spots Global Cancer Trial Database for blast phase chronic myeloid leukemia