Neoplasms

All Conditions

Rare Diseases

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neuroepithelioma

Pharmaceutical Solutions

All Drugs and Chemicals

Micronutrients

Anti-Infective Agents

Antineoplastic Agents

Iodine

Cadexomer iodine

3-Iodobenzylguanidine

Enzyme Inhibitors

Radiopharmaceuticals

This was a single arm trial, all patients were to undergo the same assessments and interventions.

Single intravenous administration of \[124I\]mIBG Solution for Injection on Day 1 with a maximum radioactive dose of 1.42 MBq/kg (±10%) and a maximum injected dose of 50 MBq \[124I\]mIBG equating to a maximum chemical dose of 10 micrograms of stable mIBG. The activity to paediatric patients will be scaled by weight based upon the EANM paediatric dose card (Lassmann et al., 2007). This will result in an activity between 10 MBq and 50 MBq depending on the patient's weight.

[124I]meta-Iodobenzylguanidine

Sue Chua, Dr

This study aims to show that 3-dimensional PET/CT imaging with a new novel PET tracer (called \[124I\]mIBG) can detect as many or more sites of neuroblastoma (a type of childhood cancer) compared to the recommended 1-dimensional routine scans (called \[123I\]mIBG planar scintigraphy).

Neuroblastoma is the most common tumour of childhood after brain tumours. Approximately half of cases are high risk and despite extensive treatments outcome is very poor. More than 60% of high risk patients suffer relapse or further spread of their disease and long-term survival is below 10%. Existing imaging techniques are not sensitive enough to accurately assess the level of risk which is critical in determining the best choice of treatment. This study will compare a new type of imaging against the existing imaging techniques. The new scans use a new tracer called \[124I\]mIBG which is taken up by the cancer tissue much more than by normal tissues. This tracer can be used with a 3D imaging technique called PET/CT to pinpoint where the disease has spread and quantify the amount of disease. Patients will be those scheduled to have an \[123I\]mIBG scan for routine care during a planned break in treatment.

A Phase I/II Study of [124I]mIBG PET/CT in Neuroblastoma

A Cancer Research UK Phase I/II Study to Compare [124I]Meta-Iodobenzylguanidine (mIBG) Positron Emission Tomography/Computerised Tomography (PET/CT) to [123I]mIBG Imaging in Patients With Metastatic Neuroblastoma

Analysis of \[124I\]mIBG PET/CT and \[123I\]mIBG planar scintigraphy was performed retrospectively by four specialist observers. The analysis was performed and observers were blinded to each others scores. The observers subsequently reviewed the lesions identified and, where there was a difference in their separate scores, they returned to the images in order to reach an agreed consensus score. The number of lesions identified as positive by this consensus scoring (all lesions with a SIOPEN score of 4 or 5) were used to meet the primary objective.

Analysis of \[124I\]mIBG PET/CT and \[123I\]mIBG SPECT/CT was performed retrospectively by four specialist observers. The analysis was performed and observers were blinded to each others scores. The observers subsequently reviewed the lesions identified and, where there was a difference in their separate scores, they returned to the images in order to reach an agreed consensus score. The number of lesions identified as positive by this consensus scoring (all lesions with a SIOPEN score of 4 or 5) were used to meet the primary objective.

Documenting Adverse Events (AEs), Serious Adverse Events (SAEs), Dose Limiting Toxicities (DLTs) (Graded According to NCI-CTCAE Version 4.02) and Laboratory Parameters and Determining Their Causality in Relation to \[123I\]mIBG and \[124I\]mIBG.

Cancer Research UK

\[124I\]meta-Iodobenzylguanidine: Single intravenous administration of \[124I\]mIBG Solution for Injection on Day 1 with a maximum radioactive dose of 1.42 MBq/kg (±10%) and a maximum injected dose of 50 MBq \[124I\]mIBG equating to a maximum chemical dose of 10 micrograms of stable mIBG. The activity to paediatric patients will be scaled by weight based upon the EANM paediatric dose card (Lassmann et al., 2007). This will result in an activity between 10 MBq and 50 MBq depending on the patient's weight.

Single Arm Trial

Age, Categorical

Sex: Female, Male

Race and Ethnicity were not collected from any participant.

Race and Ethnicity Not Collected

United Kingdom

Region of Enrollment

An exact one-sided binomial test of the null hypothesis: p ≤90% was performed using α=0.05 on the \[124I\]mIBG PET/CT imaging assessments with SIOPEN consensus scores.

Regulatory Affairs Manager

For the primary endpoint, a sensitivity analysis was also performed on patients with \<20 positive lesions on \[124I\]mIBG PET/CT to test if few patients with large numbers of positive lesions were affecting the results. The results of the sensitivity analysis were consistent with those of the overall analysis.

Secondary efficacy analysis, separate exact one-sided binomial test of the null hypothesis (p≥97%), performed (α=0.025) performed for skeletal and soft tissue lesions.

Number of positive lesions observed using [124I]mIBG PET/CT

Number of positive lesions observed using [123I]mIBG planar scintigraphy

All patients who received both \[123I\]mIBG and \[124I\]mIBG

Comparison of the Number of Lesions Detected as Positive by [123I]mIBG Planar Scintigraphy Which Are Also Considered Positive With [124I]mIBG PET/CT.

Number of positive skeletal lesions observed using [124I]mIBG PET/CT

Number of positive skeletal lesions observed using [123I]mIBG SPECT/CT

Number of positive soft tissue lesions observed using [124I]mIBG PET/CT

Number of positive soft tissue lesions observed using [123I]mIBG SPECT/CT

Comparison of the Number of Lesions Detected as Positive by [123I]mIBG SPECT Which Are Also Considered Positive With [124I]mIBG PET/CT.

All AEs

Pretreatment AEs (occurring before [123I]mIBG administration)

Treatment Emergent AEs (occurring following [123I]mIBG administration)

SAEs

[123I]mIBG Related AEs

[124I]mIBG Related AEs

Determining the Causality of Each Adverse Event to [124I]mIBG and Grading Severity According to National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE) Version 4.02.

Overall Study

Vaccines

Adjuvants, Immunologic

Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #5 (\~20 weeks), then only one 14-day cycle with each of vaccinations #6-#10.

Participants will receive oral β-glucan (40 mg/kg/day) for 14 days on, and 14 days off, beginning with vaccination #1 and continuing until vaccination #7 (\~52 weeks), then only one 14-day cycle with each of vaccinations #8-#10.

Comprised of the immunological adjuvant OPT-821 (QS-21)

OPT-821 (QS-21)

Participants will be randomized to receive this agent in two different schedules

oral β-glucan

Brian Kushner, MD

Fiorella Iglasias Cardenas, MD, MS

The purpose of this study is to test which treatment schedule of β-glucan with bivalent vaccine is more effective for participants with high-risk neuroblastoma that is in complete remission.

A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma

Phase II Trial of a Bivalent Vaccine With the Immunological Adjuvant OPT-821 (QS-21), in Combination With Randomization of Oral β-glucan, for High-Risk Neuroblastoma

Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.

To determine the effect of oral β-glucan schedule on anti-GD2 antibody titers among patients who are in first or second (or later) CR, i.e., have no evidence of neuroblastoma by standard studies.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Phase I/II Study of [124I]mIBG PET/CT in Neuroblastoma	NCT02043899	Metastatic Neur...	[124I]meta-Iodo...	1 Year -	Cancer Research UK
A Study of a Vaccine in Combination With Beta-glucan in People With Neuroblastoma	NCT06057948	Neuroblastoma High-risk Neuro... Metastatic Neur...	OPT-821 (QS-21) oral β-glucan	-	Memorial Sloan Kettering Cancer Center
A Phase I/II Study of [124I]mIBG PET/CT in Neuroblastoma	NCT02043899	Metastatic Neur...	[124I]meta-Iodo...	1 Year -	Cancer Research UK

Spots Global Cancer Trial Database for metastatic neuroblastoma