2.1 Patient Population
Patients
6-40
years of age with a histologically confirmed diagnosis of relapsed or
refractory Ewing sarcoma or high-grade osteosarcoma with evaluable or
measurable disease were eligible. Molecular evidence of translocation
involving EWSR1 or FUS was required for patients with
Ewing sarcoma. A body surface area (BSA) ≥1.25m2 and
<2m2 was required due to potential
deviations due to rounding given available size of cabozantinib tablets.
In addition, Lansky (<16 years of age) or Karnofsky (≥16 years
of age) performance score of ≥50 was required. Prior treatment with TKIs
other than cabozantinib was allowed. Prior use of topotecan and
cyclophosphamide was allowed. If this regimen immediately preceded
enrollment to the trial, then no more than two prior cycles were
allowed, and the patient must not have progressed on this combination.
Patients with unhealed and/or chronic wounds were not eligible, and
patients had to be ≥2 weeks from prior major surgical procedures.
The trial was approved by the Dana-Farber/Harvard Cancer Center
Institutional Review Board. All patients (or parent/guardian) provided
informed consent for participation.