INCB84344-102 = Ponatinib for Treatment of Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors in children [Europe]
Type of study
Safety and Efficacy of Ponatinib for Treatment of Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors in Children
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
Other trial ID
INCB 84344-102, ClinicalTrials.gov NCT03934372; EudraCT no. 2018-004878-99
What is the purpose of the study
This is a study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 year to less than 18 years with advanced leukemias, lymphomas, and solid tumors.
What will happen during the study
Key inclusion criteria
This study includes patients of all sexes who:
- are aged 1 year up to 18 years
- have a histologically or cytologically confirmed diagnosis of any of the following:
- Phase 1: Chronic myeloid leukemia in chronic phase (CP-CML), CML in blast phase (BP-CML), CML in accelerated phase (AP-CML). Acute lymphoblastic leukemia (ALL). Acute myeloid leukemia (AML). Other leukemias. Lymphoma. Any other tumors, including tumors of the central nervous system (CNS), 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 tyrosin kinase inhibitor (TKI) therapy or be in “warning” response status or have the T315I kinase domain mutation.
Patients 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: ALL. AML. Other leukemias. Lymphoma. Any other tumors, including tumors of the CNS, with specific mutations as defined in the study protocol.
Further criteria may apply. Please discuss these with your doctor or study staff.
Key exclusion criteria
Please discuss the exclusion criteria with your doctor or study staff.
Estimated primary completion date
Where can I find additional information
You can find a study description in the US register ClinicalTrials.gov. This is a database provided by the U. S. National Institutes of Health.
You can also find a study description in the EU Clinical Trials Registry. This is a database hosted by the European Medicines Agency (EMA).
Incyte Biosciences International Sàrl
Scientific lead / contact
Mohammed-El-Amine Bensmaine, MD, Incyte Biosciences International Sàrl
Study centers / principal investigators
Ghent University Hospital
The Finsen Centre National Hospital – Not yet recruiting
Hopital Robert Debre
Armand Trousseau Hospital – Not yet recruiting
Centre Hospitalier Universitaire de Poitiers
Chu de Rennes, Hospital Sud – Not yet recruiting
Universitätsklinikum Erlangen, Medizinische Klinik 5 – Not yet recruiting
Universitätsklinikum Essen – Not yet recruiting
L Azienda Ospedaliero-Universitaria Di Bologna – Not yet recruiting
Policlinico S. Orsola Malpighi
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia – Not yet recruiting
Ospedale Pediatrico G. Gaslini – Not yet recruiting
Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori
Ospedale San Gerardo, Asst Monza – Not yet recruiting
University of Milano Bicocca
Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo – Not yet recruiting
Ospedale Pediatrico Bambino Gesu Irccs
Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza
Princess Maxima Center For Pediatric Oncology
Hospital Sant Joan de Deu de Manresa – Not yet recruiting
Hospital Nino Jesus
Hospital Universitario de La Paz
Hospital Universitari I Politecnic La Fe – Not yet recruiting
Karolinska Universitetssjukhuset Solna – Not yet recruiting
Solna, 171 76
Karolinska University Hospital
University Hospital Birmingham – Not yet recruiting
Birmingham, B15 2TH
Royal Hospital For Sick Children Yorkhill Glasgow – Not yet recruiting
Alder Hey Childrens NHS Foundation Trust – Not yet recruiting
Liverpool, L12 2AP
The Royal Marsden NHS Foundation Trust
Sutton, SM2 5PT
The Royal Marsden NHS Foundation Trust – Not yet recruiting
Sutton, SW3 6JJ