NeuroblastomaPhase 2NCT02998983
Racotumomab in Patients With High-risk Neuroblastoma
A Phase 2 study testing Racotumomab in people with Neuroblastoma, run by Laboratorio Elea Phoenix S.A. at 2 sites in Argentina.
Trial summary
This clinical trial will be carried out in children diagnosed with high-risk neuroblastoma that have achieved a complete or very good partial response after standard therapy. An additional cohort of children who could not achieve these response criteria or that relapsed after standard therapy but do not have… Read more about this study
- Who is it for?
- Ages 1 year to 12 years
- Conditions the study is for
- Neuroblastoma
- 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
- 39 participants
- Where
- 2 sitesArgentina
- Study dates
- Nov 2016to Nov 2022
- Phase
- Phase 2
- 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 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed Consent or written child assent, if applicable, prior to any specific procedure of the study.
- Aged ≥ 1 year old and ≤ 12 years old at the time of diagnose.
- High-risk neuroblastoma diagnose according to the International Neuroblastoma Risk Group Staging System (INRGSS) (Annex I).
- Patients who have received complete chemotherapy, radiotherapy (if applicable) and autologous hematopoietic stem cell transplantation (if applicable) not earlier than 30 days prior to being included in the study, and patients of Group I who have completed therapy with cis retinoic acid or other maintenance onco-specific therapy using the standard dose for neuroblastoma treatment. .
- Use of concomitant metronomic chemotherapy by patients of Group II is considered acceptable.
- For patients belonging to other risk group who have relapsed or progressed, the period between the beginning of chemotherapy for the treatment of high-risk neuroblastoma and the inclusion of patients must not exceed 12 months.
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- Partial or complete remission status, very good partial remission or stable disease (pursuant to the International Neuroblastoma Response Criteria) at the time of inclusion (Annex IV).
- Assessment of the disease must be conducted within 30 days prior to inclusion.
- Additional studies supporting the response to treatment at the time of inclusion are required.
- Normal organ functions according to the following parameters:
- Adequate cardiac function as defined below:
- Electrocardiogram (ECG) 30 days prior to inclusion without substantial anomalies.
- Electrocardiogram (ECG) 30 days prior to inclusion with fractional shortening ≥27%
- Adequate bone marrow functions defined as follows:
- Neutrophil ≥1000/mm3 with no use of stimulating factor for at least 2 weeks prior to inclusion.
- Lymphocytes ≥500/mm3
- Platelets ≥ 50000/mm3.
- Adequate hepatic functions defined as follows:
- Direct bilirubin ≤1.5 x upper limit of normal (ULN)
- AST/ALT ≤ 5 x ULN
- Adequate renal function defined as follows:
- Creatinine Clearance ≥70 ml/min/1.73m2 or serum Creatinine (Cr) as per age/gender.
- Known history of Hepatitis B or C seropositivity with studies showing hepatic function results within acceptable limits may be eligible.
- Negative HIV serology.
- Pregnancy test-negative for women of childbearing potential.
- No previous Racotumomab therapy.
- No previous intravenous immunoglobulin therapy for at least one month prior to the beginning of treatment.
- Lansky Scale ≥ 50 (Annex II)
- Patients with extended bone metastasis in cranial vault or cranial base due to proximity may be considered eligible.
Who cannot join
- In order to be included, patients must not meet the following criteria:
- Neuroblastoma as progressive disease at the time of the beginning of the study.
- Patients with known hypersensitivity to any of the components of the investigational drug.
- Pregnant or breastfeeding patients.
- Patients who have received other investigational drugs or Racotumomab within 30 days prior to their inclusion in the protocol.
- History of autoimmune diseases, congenital immunodeficiencies or uncontrolled chronic diseases.
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- Acute allergy disorders or history of severe allergy reactions.
- History of demyelinating disease or inflammatory disease of the central nervous system or the peripheral nervous system.
- Patients with any of the following uncontrolled intercurrent disease:
- Active infectious diseases
- Uncontrolled cardiac disease: symptomatic congestive heart failure, serious cardiac arrhythmia.
- Known hepatic disease: cirrhosis, chronic active hepatitis or chronic persistent hepatitis.
- Convulsions not controlled with any anticonvulsant medication.
- Other malignancies after adequate therapy showing a disease-free period for more than 5 years.
- Patients receiving chronic therapy with systemic steroids and other immunosuppressive agents. Topical steroids and inhaled corticosteroids are permitted.
- History of positive HIV serology.
- Clinically symptomatic metastasis in central nervous system.
Trial locations and contacts
2 sites in 1 country.
Argentina2 sites
Prof. Dr. J. P. Garrahan National Children's Hospital
Buenos Aires, 1245
Hospital Universitario Austral
Pilar, Buenos Aires, 1629
About this study
Official title: Open-label, Multicenter, Phase II Immunotherapy Study With Racotumomab in Patients With High-risk Neuroblastoma
This clinical trial will be carried out in children diagnosed with high-risk neuroblastoma that have achieved a complete or very good partial response after standard therapy. An additional cohort of children who could not achieve these response criteria or that relapsed after standard therapy but do not have progressive disease will receive Racotumomab together with metronomic chemotherapy.
The main objectives of this study are to determine the immune response after one-year duration immunization with Racotumomab, to describe the response of Racotumomab therapy in minimal residual disease (MRD) in bone marrow and to describe the toxicity profile of Racotumomab.
Read the detailed description
Neuroblastoma is the most common extra-cranial tumor in childhood but prognosis is still poor, even with the advances in its treatment.
New therapeutic strategies have been examined, and several immunotherapeutic approaches, including combined therapy with monoclonal antibodies (anti-GD2), intravenous interleukin-2 (Il-2) and intravenous granulocyte-macrophage colony-stimulating factor (GM-CSF), 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 neuroblastoma.
The expression of the ganglioside N-glycolyl GM3 was shown in neuroblastoma and this expression could be useful as a specific target for immunotherapy.
Ractoumomab will be administered once standard therapy for neuroblastoma has been completed.
- Conditions
- Neuroblastoma
- Being tested
- Racotumomab
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RacotumomabDrug
| Study group | Role | What they receive |
|---|---|---|
| Racotumomab | Experimental | Dosage form: intradermal injection. Dosage: 0.4 mg. Frequency: the first 5 doses: biweekly injections; the following 10 doses: monthly injections. Duration: 12 months Receives: Drug: Racotumomab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants who elicit an immune response to a one-year immunization schedule of racotumomab in a cohort study of patients with high-risk neuroblastoma.
Measured: 1 year
Secondary outcomes (3)
Number of participants in whom minimally disseminated disease in the bone marrow decreases or disappears with a one-year immunization schedule of racotumomab compared to baseline values at study entry.
Measured: 1 year
Number of Participants With Treatment-Related Adverse Events as assessed by CTCAE v4.0 after a one-year immunization schedule of racotumomab when administered alone or together with onco-specific metronomic chemotherapy therapy.
Measured: 1 year
Pattern of expression of N-Glycolyl-GM3 gangliosides in tumor samples obtained at disease diagnosis and during follow up if available.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2016 (estimated)
- Study startNov 2016
- Primary completionNov 2022
- Study completionNov 2022
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Laboratorio Elea Phoenix S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Walter Cacciavillano, MD, Principal investigator, Prof. Dr. J. P. Garrahan National Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02998983
- Sponsor's protocol ID
- AR-RACO-2-16
- Status last verified
- Sep 2023
- First submitted
- 6 Dec 2016
- Last updated
- 11 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Felizzia G, Segatori VI, Galluzzo L, Alonso DF, Pinto N, Sobrero V, Varela M, Veron D, Gulino CA, Eberle SE, Fernandez D, Guthmann M, Tinelli M, Lombas C, Spitzer E, Demarco I, Montanari A, Basile C, Odella J, Posadas D, Viso M, Villar MV, Pires C, Bevilacqua G, Rubio P, Alonso C, Rossi J, Casanello AL, Rizzi A, Mascardi R, Salvador V, Ardiles N, Zubizarreta P, Cacciavillano W. Immune Response and Safety of Anti-NeuGcGM3 Anti-Idiotype Vaccine Racotumomab in Patients With High-Risk Neuroblastoma: An Open-Label, Single-Arm, Multicenter Phase 2 Study in Argentina. Pediatr Blood Cancer. 2026 Aug;73(8):e70394. doi: 10.1002/1545-5017.70394. Epub 2026 May 14.
PubMed 42136000
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Multicenter, Phase II Immunotherapy Study With Racotumomab in Patients With High-risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT02998983
- Acronym / sponsor ID
- AR-RACO-2-16
- Conditions
- Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (estimated)
- Lead sponsor
- Laboratorio Elea Phoenix S.A.Industry (pharma, biotech, devices)
- Study start
- Nov 2016 (actual)
- Primary completion
- Nov 2022 (actual)
- Study completion
- Nov 2022 (actual)
- First posted
- 21 Dec 2016 (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 clinical trial will be carried out in children diagnosed with high-risk neuroblastoma that have achieved a complete or very good partial response after standard therapy. An additional cohort of children who could not achieve these response criteria or that relapsed after standard therapy but do not have progressive disease will receive Racotumomab together with metronomic chemotherapy.
The main objectives of this study are to determine the immune response after one-year duration immunization with Racotumomab, to describe the response of Racotumomab therapy in minimal residual disease (MRD) in bone marrow and to describe the toxicity profile of Racotumomab.
Detailed description
Neuroblastoma is the most common extra-cranial tumor in childhood but prognosis is still poor, even with the advances in its treatment.
New therapeutic strategies have been examined, and several immunotherapeutic approaches, including combined therapy with monoclonal antibodies (anti-GD2), intravenous interleukin-2 (Il-2) and intravenous granulocyte-macrophage colony-stimulating factor (GM-CSF), 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 neuroblastoma.
The expression of the ganglioside N-glycolyl GM3 was shown in neuroblastoma and this expression could be useful as a specific target for immunotherapy.
Ractoumomab will be administered once standard therapy for neuroblastoma has been completed.
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: RacotumomabDosage form: intradermal injection. Dosage: 0.4 mg. Frequency: the first 5 doses: biweekly injections; the following 10 doses: monthly injections. Duration: 12 months |
Who may qualify
- Ages
- 1 year to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (29 listed)
- Informed Consent or written child assent, if applicable, prior to any specific procedure of the study.
- Aged ≥ 1 year old and ≤ 12 years old at the time of diagnose.
- High-risk neuroblastoma diagnose according to the International Neuroblastoma Risk Group Staging System (INRGSS) (Annex I).
- Patients who have received complete chemotherapy, radiotherapy (if applicable) and autologous hematopoietic stem cell transplantation (if applicable) not earlier than 30 days prior to being included in the study, and patients of Group I who have completed therapy with cis retinoic acid or other maintenance onco-specific therapy using the standard dose for neuroblastoma treatment. .
- Use of concomitant metronomic chemotherapy by patients of Group II is considered acceptable.
Key exclusion criteria (17 listed)
- Neuroblastoma as progressive disease at the time of the beginning of the study.
- Patients with known hypersensitivity to any of the components of the investigational drug.
- Pregnant or breastfeeding patients.
- Patients who have received other investigational drugs or Racotumomab within 30 days prior to their inclusion in the protocol.
- History of autoimmune diseases, congenital immunodeficiencies or uncontrolled chronic diseases.
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 |
|---|---|---|
| Racotumomab | Drug | Racotumomab |
Primary outcome
Number of participants who elicit an immune response to a one-year immunization schedule of racotumomab in a cohort study of patients with high-risk neuroblastoma.
Time frame: 1 year
3 secondary outcomes
Number of participants in whom minimally disseminated disease in the bone marrow decreases or disappears with a one-year immunization schedule of racotumomab compared to baseline values at study entry.
Time frame: 1 year
Number of Participants With Treatment-Related Adverse Events as assessed by CTCAE v4.0 after a one-year immunization schedule of racotumomab when administered alone or together with onco-specific metronomic chemotherapy therapy.
Time frame: 1 year
Pattern of expression of N-Glycolyl-GM3 gangliosides in tumor samples obtained at disease diagnosis and during follow up if available.
Time frame: 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 11 Sep 2023.
- Derived from this study: PubMed 42136000Felizzia G, Segatori VI, Galluzzo L, Alonso DF, Pinto N, Sobrero V, Varela M, Veron D, Gulino CA, Eberle SE, Fernandez D, Guthmann M, Tinelli M, Lombas C, Spitzer E, Demarco I, Montanari A, Basile C, Odella J, Posadas D, Viso M, Villar MV, Pires C, Bevilacqua G, Rubio P, Alonso C, Rossi J, Casanello AL, Rizzi A, Mascardi R, Salvador V, Ardiles N, Zubizarreta P, Cacciavillano W. Immune Response and Safety of Anti-NeuGcGM3 Anti-Idiotype Vaccine Racotumomab in Patients With High-Risk Neuroblastoma: An Open-Label, Single-Arm, Multicenter Phase 2 Study in Argentina. Pediatr Blood Cancer. 2026 Aug;73(8):e70394. doi: 10.1002/1545-5017.70394. Epub 2026 May 14.
- Individual participant dataSharing planned: No
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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
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Racotumomab in people with Neuroblastoma, run by Laboratorio Elea Phoenix S.A. at 2 sites 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 29 inclusion and 17 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 11 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Number of participants who elicit an immune response to a one-year immunization schedule of racotumomab in a cohort study of patients with high-risk neuroblastoma. (time frame: 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 NCT02998983, 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 11 Sep 2023.