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

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

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.

Eligibility Criteria:
Inclusion Criteria:

1. 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.
2. 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.
3. 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:

1. The subject was previously entered into this study.
2. The subject is pregnant, breast feeding, or menarchal and unwilling to use effective contraception during the study.
3. The subject has a history or suspicion of significant allergic reaction or anaphylaxis to any components of the 18F-mFBG imaging agent.
4. The subject is unable to lie flat or remain still for approximately 30 minutes to allow performance of a PET scan.
5. 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.