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

  • STATUS
    Recruiting
  • End date
    Jun 15, 2024
  • participants needed
    60
  • sponsor
    Incyte Biosciences International Sàrl
Updated on 26 January 2021
cancer
chronic myeloid leukemia
myeloid leukemia
lymphoid leukemia
blast crisis
lymphoma
ponatinib
leukemia
t315i mutation
blast cells
KIT
RET
solid tumour
alopecia
cns tumor

Summary

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.

Details
Condition Blast Crisis, childhood ALL, Lymphoma, leukemia, Acute myeloid leukemia, Lymphoproliferative Disorder, Lymphoma, chronic phase chronic myelogenous leukemia, Accelerated Phase Chronic Myelogenous Leukemia, Non-Hodgkin's Lymphoma, Solid Tumors, Acute Myelogenous Leukemia (AML), Solid Neoplasm, Solid Tumour, Leukemia (Pediatric), Lymphocytic Leukemia, Acute, Lymphoproliferative disorders, acute lymphoblastic leukemia, leukemia, acute lymphoblastic, leukemias, lymphomas, acute lymphoid leukaemia, acute lymphocytic leukemia, acute lymphoblastic leukemia (all), chronic phase chronic myeloid leukemia, chronic phase cml, acute myelogenous leukemia, anll, acute myeloblastic leukemia, blast phase
Treatment Ponatinib
Clinical Study IdentifierNCT03934372
SponsorIncyte Biosciences International Sàrl
Last Modified on26 January 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Histologically or cytologically confirmed diagnosis of the following malignancies: CP-CML, blast phase chronic myeloid leukemia (BP-CML), accelerated phase chronic myeloid leukemia (AP-CML); acute lymphoblastic leukemia/acute lymphocytic 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 1
Participants with CML who are resistant to or intolerant to at least 1 prior BCR-ABL-targeted TKI therapy
Participants with ALL who have failed all available or indicated therapies, which may have included 1 prior BCR-ABL-targeted TKI therapy
Participants with AML or other leukemias who have failed at least 1 prior induction attempt or for whom no effective standard therapy is available or indicated
Participants with solid tumors (including tumors of the CNS) or lymphomas who have progressed despite standard therapy or for whom no effective standard therapy is available or indicated
Phase 2 (CP-CML): Participants who are resistant to or intolerant of at least 1 prior BCR-ABL-targeted TKI therapy or have the T315I kinase domain mutation
Phase 2 (other leukemias or solid tumors)
Participants with ALL who have failed all available or indicated therapies, which must have included 1 prior BCR-ABL-targeted TKI therapy
Participants with AML or other leukemias who have failed at least 1 prior induction attempt or for whom no effective standard therapy is available or indicated
Participants with solid tumors (including tumors of the CNS) with mutation of RET, KIT, FGFR, PDGFR, VEGFR or any other mutations where ponatinib may have biological activity or lymphomas who progressed despite standard therapy or for whom no effective standard therapy is available or indicated
Karnofsky performance status 40% for participants > 16 years old or Lansky Play Scale 40 for pediatric participants 16 years old
Must have recovered to < Grade 2 per the NCI CTCAE v5.0 or to baseline from any non-hematologic toxicities (except alopecia) due to previous therapy
Willingness to avoid pregnancy or fathering children

Exclusion Criteria

Participants with CP-CML who are in MCyR or better
Prior therapies
Participants with BP-CML, ALL, or AML who have received corticosteroids or hydroxyurea within 24 hours before the first dose of ponatinib; vincristine within 7 days before the first dose of ponatinib; or other chemotherapy (excluding intrathecal chemotherapy) within 14 days before the first dose of ponatinib
Participants (except the BP-CML, ALL, and AML participants described above) who have had cytotoxic chemotherapy or radiotherapy within 21 days (or 42 days for nitrosoureas or mitomycin C) before the first dose of ponatinib
Prior radiation therapy within 6 weeks or radio-isotope therapy within 6 weeks before the first dose of ponatinib
Autologous or allogeneic stem cell transplant < 3 months before the first dose of ponatinib
Prior treatment with any of the following: immunosuppressive therapy (including post stem cell transplant regimens) within 14 days before the first dose of ponatinib; any targeted cancer therapy (including TKIs) within 7 days before the first dose of ponatinib; any other investigational anticancer agents within 30 days or 5 half-lives, whichever is longer, before randomization; any monoclonal antibody-directed anticancer therapy within 5 half-lives of the first dose of ponatinib; any chimeric antigen receptor therapy within 28 days before the first dose of ponatinib; ponatinib
Laboratory values at screening outside the protocol-defined ranges
Significant concurrent, uncontrolled medical condition, including but not limited to protocol-defined pancreatic, cardiac, cerebral, coagulation, gastrointestinal, and genetic conditions
Any active Grade 2 graft-versus-host disease
Chronic or current active uncontrolled infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment
Active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection that requires treatment or at risk for HBV reactivation
Known HIV infection
Known hypersensitivity or severe reaction to ponatinib or excipients of ponatinib
Receipt of live (including attenuated) vaccines or anticipation of need for such vaccines during the study
Females who are pregnant or lactating
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