A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta

NCT04253015 | Recruiting

• 1 other identifier: EUSA DB 0001
• Study Type: observational
• Target: 125
• Locations: 7 countries
• Sites: 13

Brief Summary

This is a non-interventional, multi-national, observational, prospective patient registry to further evaluate the effectiveness and safety of dinutuximab beta - a monoclonal immunoglobulin G 1 (IgG1) antibody, to obtain information on survival, pain severity and incidence of neuro-toxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety.

Conditions

Neuroblastoma

Keywords

Tumour; Brain tumour; Paediatrics

Outcome Measures

Primary Outcomes (4)

• Assessment of the severity of pain experienced by participants during treatment with dinutuximab beta

  Assessment of pain severity experienced by participants during the period of first dose of dinutuximab beta to the end of last 35 day course of 5th cycle of treatment

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

• Number of participants using analgesics during treatment with dinutuximab beta

  Use of analgesics during the period of first dose of dinutuximab beta to end of last 35 day course of 5th cycle of treatment

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

• Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions

  Incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions up to the end of the last 35 day course of 5th cycle of treatment

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

• Number of participants experiencing serious adverse events (SAEs) and adverse drug reactions (ADRs) during treatment with dinutuximab beta

  Number of participants experiencing serious adverse events (SAEs) and adverse drug reactions (ADRs) following the end of the last 35 day course of 5th cycle of treatment

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

Secondary Outcomes (3)

• Overall Survival (OS)

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

• Progression free survival (PFS)

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

• Event Free Survival (EFS)

  First dose of dinutuximab beta to the end of the last 35 day course of the 5th cycle of treatment (each cycle is 35 days)

Interventions

Data-collection OTHER

Data will be collected on dose, total cumulative amount of dinutuximab beta per course, dose interruptions, dose discontinuations, prophylactic treatment, use of all concomitant analgesia, assessments of pain, and occurrence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions and other AEs.

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients diagnosed with high-risk neuroblastoma who are starting treatment with dinutuximab beta in the standard clinical practice setting or participating in a clinical trial where dinutuximab beta is provided according to the indication as per the country/regional marketing authorisation, provide consent/assent and are willing to be followed up for up to 10 years. Centers who treat neuroblastoma patients with dinutuximab beta will be invited to participate in the registry. This includes networks such as the Society of Paediatric Oncology for the Treatment of Neuroblastoma (SIOPEN) in Europe.

You may qualify if:

• Patients diagnosed with high-risk neuroblastoma and starting treatment with commercially available dinutuximab beta OR
• Patients diagnosed with high-risk neuroblastoma and starting treatment with dinutuximab beta in a clinical trial where dinutuximab beta is provided according to the country/regional marketing authorisation AND
• Appropriate consent/assent has been obtained for participation in the registry with a willingness to be followed up for up to 10 years.

You may not qualify if:

• Patients commencing dinutuximab beta within a clinical trial where the product is being provided outside of the country/regional marketing authorisation OR
• Appropriate consent/assent has not been obtained for participation in the registry or patient/legal representative is not willing for the patient be followed up for up to 10 years.

Sponsors & Collaborators

• RECORDATI GROUP: lead
• United BioSource, LLC: collaborator

Study Sites (13)

St. Anna Kinderkrebsforschung

Vienna, Vienna, 1090, Austria

ACTIVE NOT RECRUITING

Centre Oscar Lambret

Lille, 59000, France

RECRUITING

Hôpital de la Timone, Hôpital des Enfants

Marseille, 13385, France

RECRUITING

Institut Curie

Paris, 75005, France

ACTIVE NOT RECRUITING

Institut Gustave Roussy

Villejuif, 94805, France

RECRUITING

Charité Berlin

Berlin, 13353, Germany

RECRUITING

Universitätsmedizin Greifswald

Greifswald, 17475, Germany

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

RECRUITING

Uniwersytecki Szpital Dziecięcy

Krakow, 30-663, Poland

RECRUITING

Hospital Universitario y Politecnico La Fe Avenida Fernando Abril Martorell

Valencia, 46026, Spain

RECRUITING

The Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, Newcastle, NE1 4LP, United Kingdom

RECRUITING

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

ACTIVE NOT RECRUITING

University Hospital Southampton

Southampton, SO16 6YD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Neuroblastoma; Neoplasms; Brain Neoplasms

Interventions

Data Collection

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Jose-Luis Garcia

  EUSA Pharma (UK) Limited

  STUDY DIRECTOR

Central Study Contacts

Jose-Luis Garcia

CONTACT

+34 663 36 34 24; PASS@eusapharma.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 10 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 27, 2020
• First Posted: February 5, 2020
• Study Start: September 30, 2019
• Primary Completion (Estimated): March 31, 2032
• Study Completion (Estimated): June 15, 2032
• Last Updated: February 7, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

FR (4); ES (1); GB (3); DE (2); AU (1); IT (1); PL (1)