Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
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).
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase 1 Study on the Use of Racotumomab Anti-idiotype Antibody in Patients With Pediatric Malignancies That Express N-glycolylated Gangliosides and Are Resistant to Conventional Treatment.|
- Selection of the higher safe dose level for ensuing clinical trials [ Time Frame: Up to 1 year ] [ Designated as safety issue: Yes ]One of the three dose levels assessed in this study will be selected for further clinical testing in children: 0.15 mg, 0,25 mg or 0.4 mg.
- Assess the immune response to racotumomab treatment [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]Active specific immunotherapy with racotumomab has shown to elicit antigen-specific immune responses in adult patients. The elicitation of anti-immunogen and anti-ganglioside antibodies will be assessed in serum samples prior and after racotumomab treatment.
|Study Start Date:||February 2011|
|Estimated Study Completion Date:||September 2014|
|Primary Completion Date:||March 2014 (Final data collection date for primary outcome measure)|
Dosage form: intradermal injection. Dosage: 0.15 mg; 0.25 mg; 0.4 mg. Frequency: 3 biweekly injections or 6 biweekly injections. Duration: 4 weeks or 10 weeks.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01598454
|Prof. Dr. J. P. Garrahan National Children's Hospital|
|Buenos Aires, Argentina, 1245|
|Principal Investigator:||Walter Cacciavillano, MD||Prof. Dr. J. P. Garrahan National Children's Hospital|
|Study Director:||Guillermo Chantada, MD||Prof. Dr. J. P. Garrahan National Children's Hospital|