INCB84344-102 = Safety and efficacy of ponatinib for recurrent or refractory leukemias or solid tumors in children [Europe]
Type of study
No longer tecruiting
INCB84344-102 = A study evaluating the safety and efficacy of ponatinib for the treatment of recurrent or refractory leukemias or solid tumors in children
What is the purpose of the study
In Phase 1, this study will determine the maximum tolerated dose and/or recommended dose for Phase 2 of oral ponatinib given once daily in pediatric participants with selected advanced hematologic malignancies or solid tumors.
In Phase 2, the study will be conducted in two groups.
Group A (CP-CML):
The aim of this study is to determine the efficacy of oral ponatinib given once daily in pediatric participants with CP-CML who are resistant or intolerant to at least 1 prior BCR-ABL–targeted TKI therapy or who have the T315I mutation.
Group B (Other Tumors):
The aim of this study is to determine the efficacy of oral ponatinib given once daily in pediatric participants with other selected advanced hematologic malignancies or solid tumors.
What will happen during the study
Key inclusion criteria
Children up to 18 years old who have histologically or cytologically confirmed diagnosis of the following malignancies:
− Chronic Phase Myeloid Chronic Leukemia (CP-CML), Blast Phase Chronic Myeloid Leukemia (BP-CML), Accelerated Phase Chronic Leukemia (AP-CML) (relapse)
− Acute Lymphoblastic Leukemia (ALL)
− Acute Myeloid Leukemai (AML)
− Other leukemias
− Any other tumors, including tumors of the central nervous system, for which standard therapy is not available or is not indicated.
Phase 2, Group A with CP-CML:
− CP-CML at the time of study entry and must be resistant to or intolerant of at least 1 prior BCR-ABL–targeted TKI therapy or have the T315I kinase domain mutation.
− Must have 1 bone marrow aspirate with documentation of BCR-ABL translocation by conventional cytogenetics, metaphase FISH, or q-PCR performed within 42 days before the first dose of ponatinib.
Phase 2, Group B with other leukemias or solid tumors:
− Other leukemias
− Any other tumors, including tumors of the central nervous system (CNS), with mutations of RET, KIT, FGFR, PDGFR, VEGFR, or any other mutations where ponatinib may have biological activity on fresh or archived tumor tissue
− Participants with solid tumors or with lymphoma must have measurable disease by computer tomograohy (CT) or magnetic resonance imageing (MRI)
Other criteria may apply.
Key exclusion criteria
Where can I find additional information
Study description in the US register ClinicalTrials.gov, a service of the U. S. National Institutes of Health
Incyte Biosciences International Sàrl
Scientific lead / contact
Peter Langmuir, MD
Study centers / principal investigators
Ghent University Hospital
Hopital Robert Debre
Centre Hospitalier Universitaire de Poitiers
Fondazione Irccs Istituto Nazionale Dei Tumori
University of Milano Bicocca
Ospedale Pediatrico Bambino Gesu IRCCS
Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza
Princess Maxima Center For Pediatric Oncology
Hospital Nino Jesus
Hospital Universitario de La Paz
Karolinska University Hospital Huddinge
Stockholm, Sweden, 14141
The Royal Marsden Nhs Foundation Trust – Sutton
Sutton, United Kingdom, SM2 5PT