"A non-randomized, open-label, multi-center, Phase I/II study of PI3K inhibitor copanlisib in pediatric patients with relapsed/refractory solid tumors or lymphoma"
Trial Description
This study is designed to investigate whether the use of copanlisib is safe, feasible and
beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or
refractory to standard therapy.
Eligibility Requirements
Inclusion Criteria:
- Signed informed consent form by patients and/or patients' parents/legal guardians and
age appropriate assent form by the patients obtained before any study specific
procedure
- Male or female patients from 6 months to ≤ 21 years old at the time of study
enrollment
- Confirmation of diagnosis:
- Phase I: Patients must have histologic verification of a solid tumor or lymphoma
malignancy at diagnosis, with measurable or evaluable disease, for which there is
no standard curative anti-cancer treatment or treatment is no longer effective
and must have received ≥ 1 prior line of therapy.
- Phase II: patients must have histologically verified tumor at initial diagnosis
and radiologically or histologically confirmed status at inclusion as indicated
in the following: neuroblastoma, osteosarcoma, rhabdomyosarcoma or Ewing sarcoma.
- Patients with solid tumors must have measurable disease (evaluable disease is
acceptable for neuroblastoma and Ewing sarcoma). Tumor assessment will be done
via computed tomography (CT), magnetic resonance imaging (MRI) or positron
emission tomography-computed tomography (PET-CT). Tumor lesions situated in a
previously irradiated area, or in an area subjected to other loco-regional
therapy, may be considered measurable if there has been demonstrated progression
in the lesion. Bone scans (if clinically indicated) should be obtained within ≤ 4
weeks prior to the start of treatment.
- Performance level: Lansky ≥ 50% for patients ≤ 16 years of age and Karnofsky ≥ 50% for
patients > 16 years of age.
- Adequate bone marrow, renal and liver function.
Exclusion Criteria:
- Active or uncontrolled infection (National Cancer Institute (NCI)-CTCAE Grade ≥ 2).
- History or concurrent condition of interstitial lung disease of any severity and/or
severely impaired lung function (as judged by the investigator).
- Diabetes mellitus.
- Uncontrolled arterial hypertension despite optimal medical management (per
institutional guidelines).
- Patients with central nervous system (CNS) malignancies.