NeuroblastomaPhase 1NCT01598454
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
A Phase 1 study testing racotumomab in people with Neuroblastoma and Ewing's Sarcoma, run by Laboratorio Elea Phoenix S.A. at 1 site in Argentina.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 10 years
- Conditions the study is for
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor
- What is being tested?
- racotumomab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteArgentina
- Study dates
- Feb 2011to Jun 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
Argentina1 site
Prof. Dr. J. P. Garrahan National Children's Hospital
Buenos Aires, 1245
About this study
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.
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.
Read more
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).
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma, Glioma
- Being tested
- racotumomab
- Medical subject headings
- Neuroblastoma, Sarcoma, Ewing, Wilms Tumor, Retinoblastoma, Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
racotumomabDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Racotumomab | Experimental | Not described Receives: Drug: racotumomab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Selection of the higher safe dose level for ensuing clinical trials
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.
Measured: Up to 1 year
Secondary outcomes (1)
Assess the immune response to racotumomab treatment
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.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2012 (estimated)
- Study startFeb 2011
- Primary completionMar 2014
- Study completionJun 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Laboratorio Elea Phoenix S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ministerio de Ciencia e Innovación, Spain
- Responsible party
- Sponsor
- Investigators
- Walter Cacciavillano, MD, Principal investigator, Prof. Dr. J. P. Garrahan National Children's HospitalGuillermo Chantada, MD, Study director, Prof. Dr. J. P. Garrahan National Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01598454
- Sponsor's protocol ID
- AR-RACO-1-2-09
- Status last verified
- Jul 2015
- First submitted
- 10 May 2012
- Last updated
- 29 Jul 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 26154941
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT01598454
- Acronym / sponsor ID
- AR-RACO-1-2-09
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma, Glioma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Laboratorio Elea Phoenix S.A.Industry (pharma, biotech, devices)
- Collaborators
- Ministerio de Ciencia e Innovación, Spain
- Study start
- Feb 2011
- Primary completion
- Mar 2014 (actual)
- Study completion
- Jun 2015 (actual)
- First posted
- 15 May 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Racotumomab | Experimental | Drug: racotumomab |
Who may qualify
- Ages
- 1 year to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| racotumomabDosage 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. | Drug | Racotumomab |
Primary outcome
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.
1 secondary outcome
Assess the immune response to racotumomab treatment
Time frame: Up to 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2015.
- Results publication: PubMed 26154941Cacciavillano 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.
- Prof. Dr. J. P. Garrahan National Children's HospitalLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Walter Cacciavillano, MD principal investigator
Prof. Dr. J. P. Garrahan National Children's Hospital
Guillermo Chantada, MD study director
Prof. Dr. J. P. Garrahan National Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing racotumomab in people with Neuroblastoma and Ewing's Sarcoma, run by Laboratorio Elea Phoenix S.A. at 1 site in Argentina.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Argentina. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Selection of the higher safe dose level for ensuing clinical trials (time frame: Up to 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01598454, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2015; AzTrials last refreshed its copy on 8 Nov 2026.