Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
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.
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 Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 1
Contacts and Locations
Study Locations
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-
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Buenos Aires, Argentina, 1245
- Prof. Dr. J. P. Garrahan National Children's Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
|---|---|
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EXPERIMENTAL: Racotumomab
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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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Selection of the higher safe dose level for ensuing clinical trials
Time Frame: 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.
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Up to 1 year
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
Assess the immune response to racotumomab treatment
Time Frame: 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.
|
Up to 1 year
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Director: Guillermo Chantada, MD, Prof. Dr. J. P. Garrahan National Children's Hospital
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ESTIMATE)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Neoplasms, Connective and Soft Tissue
- Neoplasms by Histologic Type
- Neoplasms
- Urologic Neoplasms
- Urogenital Neoplasms
- Neoplasms by Site
- Kidney Diseases
- Urologic Diseases
- Neoplasms, Glandular and Epithelial
- Eye Diseases
- Retinal Diseases
- Genetic Diseases, Inborn
- Neoplasms, Neuroepithelial
- Neuroectodermal Tumors
- Neoplasms, Germ Cell and Embryonal
- Neoplasms, Nerve Tissue
- Kidney Neoplasms
- Eye Diseases, Hereditary
- Neoplastic Syndromes, Hereditary
- Neoplasms, Complex and Mixed
- Osteosarcoma
- Neoplasms, Bone Tissue
- Neoplasms, Connective Tissue
- Sarcoma
- Neuroectodermal Tumors, Primitive
- Neuroectodermal Tumors, Primitive, Peripheral
- Eye Neoplasms
- Retinal Neoplasms
- Sarcoma, Ewing
- Neuroblastoma
- Retinoblastoma
- Wilms Tumor
Other Study ID Numbers
- AR-RACO-1-2-09
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