A Post-Authorisation Safety Study Patient Registry of Patients With Neuroblastoma Being Treated With Dinutuximab Beta
NCT ID: NCT04253015
Last Updated: 2024-02-07
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
125 participants
OBSERVATIONAL
2019-09-30
2032-06-15
Brief Summary
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Detailed Description
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Neuroblastoma, is the most common extra-cranial solid tumour in children. Most patients with neuroblastoma are diagnosed under the age of 5 years and most present with metastatic disease and/or high-risk features. Despite the introduction of novel treatment strategies, including high-dose chemotherapy followed by autologous stem cell transplantation (ASCT), the outcome of these patients remains poor.
Dinutuximab beta is a chimeric monoclonal immunoglobulin G 1 (IgG1) antibody that is specifically directed against the carbohydrate moiety of disialoganglioside antigen (GD2), which is overexpressed on neuroblastoma cells. By binding to neuroblastoma cells, dinutuximab beta can induce both complement dependent cytotoxicity (CDC) and antibody dependent cell-mediated cytotoxicity (ADCC).
The efficacy of dinutuximab beta has been evaluated in a randomised controlled trial comparing the administration of dinutuximab beta with or without interleukin 2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two singlearm studies in the relapsed/refractory setting. The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on survival, pain severity and incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety.
Study Design:
This is a non-interventional, multi-national, observational, prospective patient registry of patients with high-risk neuroblastoma being treated with the monoclonal antibody dinutuximab beta. The efficacy and safety of dinutuximab beta will further be evaluated in this registry that will provide information on survival, pain severity and incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, hypersensitivity reactions and long-term safety.
Research Questions and Objectives:
Primary objectives:
* To assess pain severity and use of analgesics during the period of first dose of dinutuximab beta to end of last 35 day course of the 5th cycle of treatment
* To assess the incidence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events and hypersensitivity reactions
* To assess the long term safety profile
Secondary objectives:
* Progression free survival (PFS) in patients treated with dinutuximab beta.
* Event Free Survival (EFS) in patients treated with dinutuximab beta
* Overall survival (OS) in patients treated with dinutuximab beta
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.
Study Size:
It is planned to enroll a sufficient number of patients (estimated at 125) such that 100 patients will have completed all five treatment courses of dinutuximab beta. It is anticipated that this will result in 40-50 patients who are progression free at 10 years.
Data Sources:
Data will be collected from physicians using an electronic data capture (EDC) system. The electronic case report forms (eCRFs) will be designed to gather data from the medical records at baseline, during treatment and at normal clinical practice follow up visits.
Data Analysis:
The safety analysis set, containing all patients treated with at least one dose of dinutuximab beta will be considered for safety and efficacy analyses. All baseline, treatment period and follow up characteristics will be summarized using descriptive statistics. Endpoints addressing primary and secondary analysis will include 95% confidence intervals (CIs) including the Clopper Pearson method for binomial, log-log transform for survival. OS, PFS and EFS will be analysed using Kaplan-Meier methods.
Variables:
Baseline (prior to start of treatment): Demographics, clinical trial participation, neuroblastoma disease history, and presence or absence of neurotoxicity, visual impairment, and cardiovascular abnormality.
Treatment period (up to end of last 35 day course of 5th cycle of treatment):
Dosing regimen, total cumulative amount of dinutuximab beta per course, concomitant medications during course (IL-2, retinoic acid and/or antihistamines), daily analgesics (opioids, gabapentin/ pregabalin and/or non-opioid analgesics and other neuropathic pain treatments), daily pain assessment during infusion of dinutuximab beta, occurrence of neurotoxicity, visual impairment, capillary leak syndrome, cardiovascular events, and hypersensitivity reactions, adverse events (AEs)/serious adverse events (SAEs) treatment interruptions and discontinuations, progression of disease, date and cause of death, reason for study withdrawal (if applicable) Follow up (from end of last 35 day course of 5th cycle of treatment): Status of neurotoxicity, visual impairment, cardiovascular events, (resolved, not resolved), SAEs and adverse drug reaction (ADRs), progression of disease, date and cause of death, reason for study withdrawal (if applicable).
Conditions
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Study Design
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COHORT
PROSPECTIVE
Interventions
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Data-collection
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
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
1 Year
18 Years
ALL
No
Sponsors
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United BioSource, LLC
INDUSTRY
RECORDATI GROUP
INDUSTRY
Responsible Party
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Principal Investigators
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Jose-Luis Garcia
Role: STUDY_DIRECTOR
EUSA Pharma (UK) Limited
Locations
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St. Anna Kinderkrebsforschung
Vienna, Vienna, Austria
Centre Oscar Lambret
Lille, , France
Hôpital de la Timone, Hôpital des Enfants
Marseille, , France
Institut Curie
Paris, , France
Institut Gustave Roussy
Villejuif, , France
Charité Berlin
Berlin, , Germany
Universitätsmedizin Greifswald
Greifswald, , Germany
IRCCS Istituto Giannina Gaslini
Genova, , Italy
Uniwersytecki Szpital Dziecięcy
Krakow, , Poland
Hospital Universitario y Politecnico La Fe Avenida Fernando Abril Martorell
Valencia, , Spain
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Newcastle, United Kingdom
Birmingham Children's Hospital
Birmingham, , United Kingdom
University Hospital Southampton
Southampton, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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Sylvie Abed
Role: primary
Desiree Ramal
Role: primary
Other Identifiers
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EUSA DB 0001
Identifier Type: -
Identifier Source: org_study_id
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