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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Neuroblastoma
• Intervention / Treatment: Drug: 18F-MFBG

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 43
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: A F Jacobson, MD, PhD; Phone Number: 401-375-2686; Email: Jacobson@innervaterp.com

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
    • San Francisco, California, United States, 94143
      • University of California, San Francisco
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children - Indiana University
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Health System
  • New York
    • New York, New York, United States, 10065
      • Memorial Sloan Kettering Cancer Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • The Children's Hospital of Philadelphia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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.

Collaborators and Investigators

• Sponsor: Innervate Radiopharmaceuticals LLC (Formerly: Illumina Radiopharmaceuticals LLC)
• Collaborators: Memorial Sloan Kettering Cancer Center; New Approaches to Neuroblastoma Therapy Consortium

Study record dates

Study Major Dates

• Study Start (Actual): November 18, 2021
• Primary Completion (Actual): February 1, 2024
• Study Completion (Estimated): May 1, 2024

Study Registration Dates

• First Submitted: January 18, 2021
• First Submitted That Met QC Criteria: January 21, 2021
• First Posted (Actual): January 26, 2021

Study Record Updates

• Last Update Posted (Actual): February 13, 2024
• Last Update Submitted That Met QC Criteria: February 9, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: 18F-mFBG; meta-fluorobenzylguanidine
• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma
• Other Study ID Numbers: IRP101-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No