Racotumomab in Patients With High-risk Neuroblastoma

NCT02998983 | Completed Phase 2

• 1 other identifier: AR-RACO-2-16
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 2

Brief 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 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.

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• 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.

  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.

  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.

  1 year

• Pattern of expression of N-Glycolyl-GM3 gangliosides in tumor samples obtained at disease diagnosis and during follow up if available.

  1 year

Study Arms (1)

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

Drug: Racotumomab

Interventions

Racotumomab DRUG

Racotumomab

Eligibility Criteria

• Age: 1 Year - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Normal organ functions according to the following parameters:
• Adequate cardiac function as defined below:
• Adequate bone marrow functions defined as follows:
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Laboratorio Elea Phoenix S.A.: lead

Study Sites (2)

Hospital Universitario Austral

Pilar, Buenos Aires, 1629, Argentina

Location

Prof. Dr. J. P. Garrahan National Children's Hospital

Buenos Aires, 1245, Argentina

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

racotumomab

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Walter Cacciavillano, MD

  Prof. Dr. J. P. Garrahan National Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 6, 2016
• First Posted: December 21, 2016
• Study Start: November 1, 2016
• Primary Completion: November 1, 2022
• Study Completion: November 1, 2022
• Last Updated: September 11, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

AR (2)