INCB84344-102 = Ponatinib for Treatment of Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors in children [Europe]

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

This study is conducted in two phases.
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 Phase 2, ponatinib is administered at the recommended Phase 2 dose.

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).

Study centers / principal investigators

Belgium

Ghent University Hospital
Ghent, 09000

Universitair Ziekenhuis (Uz) Leuven – Not yet recruiting
Leuven, 030000

Denmark

The Finsen Centre National Hospital – Not yet recruiting
Copenhagen, 02100

France

Hopital Robert Debre
Paris, 75019

Armand Trousseau Hospital
Paris, 75571

Centre Hospitalier Universitaire de Poitiers
Poitiers, 86021

Chu de Rennes, Hospital Sud
Rennes, 35700

Germany

Universitätsklinikum Erlangen, Medizinische Klinik 5 – Not yet recruiting
Erlangen, 91054

Universitätsklinikum Essen – Not yet recruiting
Essen, 45147

Italy

Azienda Ospedaliero-Universitaria Di Bologna
Policlinico S. Orsola Malpighi
Bologna, 40138

Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Brescia, 25123

Ospedale Pediatrico G. Gaslini
Genova, 16147

Comitato Etico Fondazione Irccs Istituto Nazionale Dei Tumori Milano
Milan, 20133

Ospedale San Gerardo, Asst Monza – Not yet recruiting
Monza, 20900

University of Milano Bicocca
Monza, 20900

Aorn Santobono Pausilipon – Not yet recruiting
Naples, 80122

Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo – Not yet recruiting
Pavia, 27100

Ospedale Pediatrico Bambino Gesu Irccs
Rome, 00165

Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza
Torino, 10126

The Netherlands

Princess Maxima Center For Pediatric Oncology
Utrecht, 03584

Spain

Hospital General Universitario Vall D Hebron – Not yet recruiting
Barcelona, 08035

Hospital Sant Joan de Deu de Manresa – Not yet recruiting
Barcelona, 08035

Hospital Infantil Universitario Nino Jesus
Madrid, 28009

Hospital Universitario de La Paz
Madrid, 28046

Hospital Universitari I Politecnic La Fe – Not yet recruiting
Valencia, 46026

Sweden

Karolinska Universitetssjukhuset Solna – Not yet recruiting
Solna, 171 76

Karolinska University Hospital
Stockholm, 14141

United Kingdom

University Hospital Birmingham – Not yet recruiting
Birmingham, B15 2TH

Royal Hospital For Sick Children Yorkhill Glasgow – Not yet recruiting
Glasgow, G514TF

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