Racotumomab in Patients With High-risk Neuroblastoma

• Register: ClinicalTrials.gov
• Last refreshed on: 18 September 2023
• Main ID: NCT02998983
• Date of registration: 06/12/2016
• Prospective Registration: No
• Primary sponsor: Laboratorio Elea Phoenix S.A.
• Public title: Racotumomab in Patients With High-risk Neuroblastoma
• Scientific title: Open-label, Multicenter, Phase II Immunotherapy Study With Racotumomab in Patients With High-risk Neuroblastoma
• Date of first enrolment: November 2016
• Target sample size: 39
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02998983
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Argentina

Contacts

• Name: Walter Cacciavillano, MD
• Affiliation: Prof. Dr. J. P. Garrahan National Children's Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Informed Consent or written child assent, if applicable, prior to any specific
procedure of the study.

2. Aged = 1 year old and = 12 years old at the time of diagnose.

3. High-risk neuroblastoma diagnose according to the International Neuroblastoma Risk
Group Staging System (INRGSS) (Annex I).

4. 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. .

5. Use of concomitant metronomic chemotherapy by patients of Group II is considered
acceptable.

6. 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.

7. 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).

8. Assessment of the disease must be conducted within 30 days prior to inclusion.

9. Additional studies supporting the response to treatment at the time of inclusion are
required.

10. 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.

11. Known history of Hepatitis B or C seropositivity with studies showing hepatic function
results within acceptable limits may be eligible.

12. Negative HIV serology.

13. Pregnancy test-negative for women of childbearing potential.

14. No previous Racotumomab therapy.

15. No previous intravenous immunoglobulin therapy for at least one month prior to the
beginning of treatment.

16. Lansky Scale = 50 (Annex II)

17. Patients with extended bone metastasis in cranial vault or cranial base due to
proximity may be considered eligible.

Exclusion Criteria:

In order to be included, patients must not meet the following criteria:

1. Neuroblastoma as progressive disease at the time of the beginning of the study.

2. Patients with known hypersensitivity to any of the components of the investigational
drug.

3. Pregnant or breastfeeding patients.

4. Patients who have received other investigational drugs or Racotumomab within 30 days
prior to their inclusion in the protocol.

5. History of autoimmune diseases, congenital immunodeficiencies or uncontrolled chronic
diseases.

6. Acute allergy disorders or history of severe allergy reactions.

7. History of demyelinating disease or inflammatory disease of the central nervous system
or the peripheral nervous system.

8. 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.

9. Other malignancies after adequate therapy showing a disease-free period for more than
5 years.

10. Patients receiving chronic therapy with systemic steroids and other immunosuppressive
agents. Topical steroids and inhaled corticosteroids are permitted.

11. History of positive HIV serology.

12. Clinically symptomatic metastasis in central nervous system.



Age minimum: 1 Year
Age maximum: 12 Years
Gender: All

Health Condition(s) or Problem(s) studied

Neuroblastoma

Intervention(s)

Drug: Racotumomab

Primary Outcome(s)

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]

Secondary Outcome(s)

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]

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]

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]

Secondary ID(s)

AR-RACO-2-16

Source(s) of Monetary Support

Please refer to primary and secondary sponsors