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

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.

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.