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

STATUS

Recruiting
End date

Jan 4, 2025
participants needed

60
sponsor

Incyte Biosciences International Sàrl

Send

Updated on 22 October 2022

See if I qualify

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	Acute Myeloid Leukemia, Accelerated Phase Chronic Myeloid Leukemia, Blast Phase Chronic Myeloid Leukemia, Chronic Phase Chronic Myeloid Leukemia, Acute Lymphoblastic Leukemia, Acute Lymphocytic Leukemia, Leukemia, Lymphoma, Solid Tumors
Treatment	Ponatinib
Clinical Study Identifier	NCT03934372
Sponsor	Incyte Biosciences International Sàrl
Last Modified on	22 October 2022

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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
	Phase 2 (CP-CML): Participants who are resistant to or intolerant of at least 1 prior
	BCR-ABL-targeted TKI therapy
	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
	Participants with ALL who have all available or indicated therapies, which must have included 1 prior BCR-ABL-targeted TKI therapy
	Phase 2 (other leukemias or solid tumors)
	Participants with solid tumors (including tumors of the CNS) with 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
	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
	Karnofsky performance status
	Willingness to avoid pregnancy or fathering children
Exclusion Criteria
	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 within 21 days (or 42 days for nitrosoureas or mitomycin C) 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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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