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

Sponsor

Laboratorio Elea Phoenix S.A. (Industry)

Overall Status

Completed

CT.gov ID

NCT01598454

Collaborator

Ministerio de Ciencia e Innovación, Spain (Other)

Enrollment

Location

Arm

51.9

Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief 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).

Condition or Disease	Intervention/Treatment	Phase
Neuroblastoma Ewing's Sarcoma Wilm's Tumor Retinoblastoma Glioma	Drug: racotumomab	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

15 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (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.

Study Start Date :

Feb 1, 2011

Actual Primary Completion Date :

Mar 1, 2014

Actual Study Completion Date :

Jun 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Racotumomab	Drug: racotumomab 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.

Arm

Intervention/Treatment

Experimental: Racotumomab

Drug: racotumomab

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.

Outcome Measures

Primary Outcome Measures

Selection of the higher safe dose level for ensuing clinical trials [Up to 1 year]
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.

Secondary Outcome Measures

Assess the immune response to racotumomab treatment [Up to 1 year]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 10 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Children (both genders) of between 1 and 10 years old at the time of accrual.
Diagnosis of neuroblastoma (progression after first line treatment), glioma (progressing disease or metastatic disease, without curative treatment options), Ewing's sarcoma (progressive metastatic disease to first line treatment or progressive disease to second line treatment), Wilm's tumor (metastatic relapse after treatment), or retinoblastoma (progressing disease or metastatic relapse during or after first line treatment).
Previous cancer treatment finished 30 days before accrual.
Lansky performance status over 50.

Exclusion Criteria:

History of encephalopathy, convulsions, asthma or severe allergy.
Infectious disease grade 3 or 4 according to CTCAE version 3.
Hepatic, kidney or cardiac insufficiency.
Marrow insufficiency after self-transplantation of hematopoietic stem cells.
Weight inferior to 12 kg at the time of accrual.
Concomitant cancer treatment.
Inability to comply with study procedures.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Prof. Dr. J. P. Garrahan National Children's Hospital	Buenos Aires		Argentina	1245

Sponsors and Collaborators

Laboratorio Elea Phoenix S.A.
Ministerio de Ciencia e Innovación, Spain

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.

Responsible Party:

Laboratorio Elea Phoenix S.A.

ClinicalTrials.gov Identifier:

NCT01598454

Other Study ID Numbers:

AR-RACO-1-2-09

First Posted:

May 15, 2012

Last Update Posted:

Jul 29, 2015

Last Verified:

Jul 1, 2015

Additional relevant MeSH terms:

Study Results

No Results Posted as of Jul 29, 2015