Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides

Summary

This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy. The acute toxicity and immune response will be evaluated. The expression of N-glycolylated gangliosides in tumors has previously been investigated in the tumor sample bank at this Hospital. The expression of N-glycolyl GM3 was shown in neuroblastoma, Ewing's sarcoma, Wilm's tumor and retinoblastoma. Gliomas and the aforementioned tumor types have a very bad prognosis when conventional treatment is ineffective. New therapeutic strategies have thus been examined, and several immunotherapeutic approaches, including dendritic cell vaccines, peptide vaccines and anti-idiotype vaccines are currently being assessed. Racotumomab is an anti-idiotype antibody capable of inducing anti-N-glycolyl GM3 antibodies in patients with melanoma, breast cancer and lung cancer. Dose escalation studies have shown the safety of racotumomab in the 0.5 to 2 mg dose range. The 1 mg dose level was selected for the ensuing clinical studies. This clinical trial in children involves three dose levels: 0.15 mg, 0.25 mg and 0.4 mg, owing to the difference in body surface between an adult (1.73 sq. m in average) and the candidate population for this study (0.55 to 0.7 sq. m).

Timeline

Start

2011-02

Primary completion

2014-03

Completion

2015-06

Publications

• Results Cacciavillano W, Sampor C, Venier C, Gabri MR, de Davila MT, Galluzzo ML, Guthmann MD, Fainboim L, Alonso DF, Chantada GL. A Phase I Study of the Anti-Idiotype Vaccine Racotumomab in Neuroblastoma and Other Pediatric Refractory Malignancies. Pediatr Blood Cancer. 2015 Dec;62(12):2120-4. doi: 10.1002/pbc.25631. Epub 2015 Jul 7.

Drugs

Indications