An Imaging Study of Anti-GD2-800CW in Patients With Neuroblastoma
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: anti-GD2-800CW.
- Who it may be relevant to
- Registry conditions: Neuroblastoma. Basic parameters: 1 year — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Netherlands
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase Ib/II First-in-human Imaging Study of Anti-GD2-800CW in Patients With Neuroblastoma
Overview
This is a non-randomized, open label phase Ib/II dose-escalation and expansion study designed to define the recommended dose of anti-GD2-800CW in pediatric neuroblastoma patients. The aim of this imaging study is to determine a safe and effective dose of anti-GD2-800CW for intra-operative detection of neuroblastoma using NIR fluorescence.
Detailed description
Neuroblastoma is the most commonly diagnosed extracranial solid tumor in young children. It is a neural crest derived malignancy, which are the cells that give rise to the sympathetic nervous system, and can therefore be located in different body compartments. However, it occurs most frequently (80%) in the abdomen. Current treatment involves debulking surgery using standard-of-care, not assisted by intra-operative visualization of the tumor. In this current situation there is, i) a high risk of severe complications such as bleeding or unplanned nephrectomy (40%), ii) a high chance of \>10% residual tumor tissue, and iii) difficulty distinguishing neuroblastoma subtypes with varying malignancy. Therefore, there is a high need for new tools enabling tumor-specific intra-operative visualization to assist the surgeon. This will reduce surgical complications and ensure neuroblastoma cells can be visualized and resected during surgery. Over the past years, Fluorescence Guided Surgery (FGS) has proven to be a useful intraoperative tool to increase the visibility of the tumor in various adult cancers. Our group has developed and validated a tumor-specific fluorescent agent, anti-GD2-800CW, that can be used to visualize neuroblastoma during surgery. Anti-GD2-800CW consists of two substances. Firstly, the monoclonal antibody anti-GD2, also known as Qarziba or Dinutuximab-beta, which binds to Disialoganglioside 2 (GD2), a tumor-associated antigen specifically expressed by neuroblastoma, most melanomas and several other tumors. Immunotherapy with anti-GD2 is now standard therapy for high-risk neuroblastoma treatment in patients aged 12 months and above. Secondly, the near-infrared (NIR) fluorescent dye, IRDye800CW, which is used extensively in clinical trials as fluorescent imaging agent without any known adverse events (AEs). The aim of this study is to investigate the safety and efficacy of this novel agent, anti-GD2-800CW, to visualize neuroblastoma during surgery.
Interventions
- Drug anti-GD2-800CW
Participants will receive a single dose of anti-GD2-800CW 96 hours prior to surgery. In phase Ib, using a 3+3 design, a dose escalation is performed to determine the RP2D.
Primary outcome measures
- To establish the recommended phase 2 dose (RP2D), based on efficacy and safety, of the anti-GD2-800CW fluorescent imaging agent in pediatric patients with neuroblastoma. [Time frame: Through escalation phase completion, an average of 1 year.]
- To investigate safety of anti-GD2-800CW based on treatment related new AEs. [Time frame: From IMP administration to 4 days after surgery]
Secondary outcome measures (5)
- To assess the pharmacodynamic characteristics of an i.v. injection of anti-GD2-800CW. [Time frame: From just before infusion to 4 days after surgery.]
- To assess the pharmacokinetic characteristics of an i.v. injection of anti-GD2-800CW. [Time frame: From just before infusion to 4 days after surgery.]
- To correlate clinical imaging data with histopathology data to determine the accuracy for depicting GD-2 expressing vital tumor cells. [Time frame: From start surgery to 3 weeks after surgery when the PA results are analyzed.]
- To assess the intra-operative usability of anti-GD2-800CW to distinguish tumor tissue from surrounding tissue on eyesight of the surgeons. [Time frame: From start surgery to 3 weeks after surgery when the PA results are analyzed.]
- To assess whether tumor load influences the fluorescence intensity. [Time frame: From pre-operative scan to when PA results are analyzed, 3 weeks after surgery.]
Eligibility criteria
Inclusion criteria
- Patients with the diagnosis of neuroblastoma as defined by histopathology (confirmed by the PMC Department of Pathology), who will be operated for NB as standard of care procedure
- Patients older than 1 year of age and not older than 18 years.
- Written informed consent from patients and/or from parents or legal guardians, according to local law and regulations
Exclusion criteria
- Previous treatment with Dinutuximab-beta, either alone or in combination with chemotherapy
- Pregnancy or positive pregnancy test (urine or serum) in females of childbearing potential. Pregnancy test must be performed within during the screening period and before the administration of the IMP.
- Breast feeding.
- Sexually active participants not willing to use highly effective contraceptive method (pearl index <1) as defined in CTFG HMA 2020 (Appendix I) during trial participation and until 6 months after end of protocol therapy.
- Patients that received prior treatment with chimeric antibodies.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Netherlands · 1 center
- Prinses Maxima Centrum — Utrecht
Identifiers
NCT: NCT07399821 · 2023-507596-22-00 · 2023-507596-22-00