Phase II study of ganitumab, a fully human anti-type-1 insulin-like growth factor receptor antibody, in patients with metastatic ewing family tumors or desmoplastic small round cell tumors

Journal of Clinical Oncology, 04/25/2012

Tap WD et al. – Ganitumab was well tolerated and demonstrated antitumor activity in patients with advanced recurrent EFT or DSRCT.


  • Patients ?16 years of age with relapsed or refractory EFT or DSRCT received 12 mg/kg of ganitumab every 2 weeks.
  • Objective response rate (ORR) was the primary end point.
  • Secondary end points included clinical benefit rate (CBR = complete + partial responses + stable disease [SD] ? 24 weeks) and safety and pharmacokinetic profiles of ganitumab.
  • The relationship between tumor response and EWS gene translocation status and IGF-1 levels was evaluated.


  • Thirty-eight patients (22 with EFT; 16 with DSRCT) received one or more doses of ganitumab.
  • Twenty-four patients (63%) experienced ganitumab-related adverse events.
  • Grade 3 related events included hyperglycemia (n = 2), thrombocytopenia (n = 5), neutropenia (n = 2), leukopenia (n = 1), and transient ischemic attack (n = 1).
  • There were no grade 4 or 5 treatment-related events. Of 35 patients assessed for response, two had partial responses (ORR, 6%) and 17 (49%) had SD.
  • Four patients had SD ? 24 weeks, contributing to a CBR of 17%.
  • The pharmacokinetic profile of ganitumab was similar to that observed in the first-in-human trial. Elevation of IGF-1 levels was observed postdose.
  • EWS-Fli1 translocations were analyzed by RNA sequencing and fluorescent in situ hybridization, and novel translocations were observed in EFT and DSCRT.
  • No apparent relationship between tumor response and IGF-1 levels or EWS gene translocations was observed.

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