INCB84344-102 = Safety and efficacy of ponatinib for recurrent or refractory leukemias or solid tumors in children [Europe]

Type of study

Pediatric trials

Current status

No longer tecruiting

Study title

INCB84344-102 = A study evaluating the safety and efficacy of ponatinib for the treatment of recurrent or refractory leukemias or solid tumors in children

Scientific title

An Open-Label, Single-Arm, Phase 1/2 Study Evaluating the Safety and Efficacy of Ponatinib for the Treatment of Recurrent or Refractory Leukemias or Solid Tumors in Pediatric Participants (ClinicalTrials.gov no NCT03934372, EudraCT no. 2018-004878-99)

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:

Phase 1:
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
− Lymphoma
− 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:
− ALL
− AML
− Other leukemias
− Lymphoma
− 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

Study sponsor

Incyte Biosciences International Sàrl
Switzerland

Scientific lead / contact

Peter Langmuir, MD
Incyte Corporation
globalmedinfo@incyte.com

Principal investigator

Multiple

Study centers / principal investigators

Belgium

Ghent University Hospital
Gent, 9000


France

Hopital Robert Debre
Paris, 75019

Centre Hospitalier Universitaire de Poitiers
Poitiers, 86021

Italy

Fondazione Irccs Istituto Nazionale Dei Tumori
Milano, 20133

University of Milano Bicocca
Monza, 20900

Ospedale Pediatrico Bambino Gesu IRCCS
Roma, 00165

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

Netherlands

Princess Maxima Center For Pediatric Oncology
Utrecht, 03584

Spain

Hospital Nino Jesus
Madrid, 28009

Hospital Universitario de La Paz
Madrid, 28046


Sweden

Karolinska University Hospital Huddinge
Stockholm, Sweden, 14141


United Kingdom

The Royal Marsden Nhs Foundation Trust – Sutton
Sutton, United Kingdom, SM2 5PT