Open-Label Study of 18F-mFBG for Imaging Neuroblastoma

Study Overview
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Brief Summary
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This is a Phase 3 study evaluating the positron-emitting radiopharmaceutical 18F-mFBG as an imaging agent for confirming or excluding the presence of neuroblastoma

Detailed Description
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This is a prospective, Phase 3, multi-center, open-label study designed to assess the use of 18F-mFBG PET imaging in subjects with known or presumed neuroblastoma. Eligible participants will have either histopathologically established diagnosis of neuroblastoma or a presumed diagnosis based on signs, symptoms, physical examination, imaging findings, and laboratory and genetic test results. Subjects will only be administered 18F-mFBG if they have undergone or are scheduled to undergo a 123I-mIBG or 18F-FDG scan within 30 days of the date of the 18F-mFBG scan. Subjects should not have received any chemotherapy, immunotherapy or radiotherapy between the clinical and 18F-mFBG imaging studies. 18F-mFBG PET studies and clinical 123I-mIBG scans will be evaluated off-site by 3 independent radiologists and/or nuclear medicine physicians who are blinded to all subject information. Blinded readers will record whether findings on 18F-mFBG and 123I-mIBG scans are consistent with presence of neuroblastoma (Disease +) or absence of disease (Disease -). Subject-level diagnostic performance will be assessed on the basis of positive and negative percent agreement (PPA and NPA) between the blinded 18F-mFBG scan interpretations and disease status (+ or - for neuroblastoma) as independently assessed by an Expert Panel of pediatric oncologists. Secondary efficacy analyses will compare PPA and NPA between 18F-mFBG and 123I-mIBG interpretations provided by the blinded readers.

Official Title
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A Prospective Phase 3 Multi-center Study to Assess the Efficacy and Safety of 18F-mFBG PET Imaging in Subjects With Neuroblastoma

Conditions
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Neuroblastoma

Intervention / Treatment
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* Drug: 18F-MFBG


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: a) An established diagnosis of neuroblastoma based on unequivocal histopathology from tissue obtained at any time prior to enrolment in the trial; OR b) A presumed diagnosis of neuroblastoma based on signs, symptoms, physical examination, imaging findings, and laboratory and genetic test results, including individuals in whom therapy has already been empirically initiated. The subject has undergone or is scheduled to undergo evaluation of neuroblastoma disease status, including at least one functional imaging study (123I-mIBG or 18F-FDG), within 30 days prior to the date of 18F-mFBG imaging, with no interval therapy between such evaluation and investigational drug administration. Ability of subject or subject's legal guardian to understand and sign a written informed consent document, including, for subjects age 7-17, an assent form. Exclusion Criteria: The subject was previously entered into this study. The subject is pregnant, breast feeding, or menarchal and unwilling to use effective contraception during the study. The subject has a history or suspicion of significant allergic reaction or anaphylaxis to any components of the 18F-mFBG imaging agent. The subject is unable to lie flat or remain still for approximately 30 minutes to allow performance of a PET scan. The subject uses medications that are known to interfere with uptake of NET-dependent agents and these medications cannot be safely withheld 24 hours before study procedures.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Intervention Model: Single Group Assignment
Interventional Model Description: Comparison of PET imaging interpretation and expert assessment for presence or absence of neuroblastoma tumor
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Study Cohort: Subjects with known or presumed neuroblastoma<br>Drug: 18F-mFBG Positron-emitting radiopharmaceutical 18F-mFBG as an imaging agent for identification of neuroblastoma. Other Names: meta-fluorobenzylguanidine IRP101 | Drug: 18F-MFBG<br>* Positron-emitting tomography (PET) (either PET/CT or PET/MR) imaging agent<br>* Other names: IRP101;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| PET Scan Identification of Neuroblastoma | Focal 18F-mFBG uptake (presence or absence) when imaging at 60 minutes with PET/CT or PET/MR, reported as positive and negative predictive agreement with expert oncologist assessment for presence of neuroblastoma | Imaging at 60 minutes | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Comparison of 18F-mFBG PET With Clinical 123I-mIBG Imaging | This objective will be assessed using the secondary endpoint of focal increased 123I-mIBG uptake (presence or absence), reported as positive and negative predictive agreement with expert oncologist assessment for presence of neuroblastoma | Scan interpretation up to one month after intervention. | 
| Number of subjects with adverse events graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 5. | Description: Assessments based on changes between pre- and post-18F-mFBG administration physical examination findings, vital signs measurements, blood test results including hematology and biochemistry, and electrocardiograms (ECGs). | 24 hours following 18F-mFBG administration. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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18F-mFBG, meta-fluorobenzylguanidine