13 Months and older, any sex, with Neuroblastoma or Medulloblastoma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Determine Feasibility Using Days From the Date of Biopsy to Date of Start of TreatmentPrimary· Date from biopsy to completion of 1 cycle of therapy, generally about 30 days
Days to treatment is one data point that will be used in order to determine feasibility. The definition of feasibility for this study will include: "Enrollment onto study, RNA expression profile completed, DNA Mutation Panel completed, genomic analysis and report generation, tumor board held with treatment decision, treatment review completed and start of treatment by 21 days post biopsy/surgical resection date, and then completion of 1 cycle of therapy."
Group
Value
95% CI
Stratum 1
12.2
7 – 21
Stratum 2
19.9
14 – 30
Stratum 3
14.9
9 – 29
Number of Participants With Adverse Events as a Measure of SafetySecondary· Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years.
To determine the safety of allowing a molecular tumor board to determine individualized treatment plans
Group
Value
95% CI
Guided Therapy- Pediatric Gene Analysis Platform
43
Overall Response Rate (ORR) of Participants by the Presence of Radiologically Assessable Disease by Cross-sectional CT or MRI Imaging and/or by MIBG or PET Scans.Secondary· Followed until off therapy, generally 3 years
To determine the activity of treatments chosen based on Overall response rate (ORR) using RESIST criteria. The assessment of response will include the initial measurable targets and will be performed after cycle 2, then after every other cycle. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by CT or MRI imaging and/or by MIBG or PET scans: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease, At least a 20% increase in
Group
Value
95% CI
Stratum 1
1
Stratum 2
2
Stratum 3
2
Stratum 1
0
Stratum 2
0
Stratum 3
1
Stratum 1
4
Stratum 2
0
Stratum 3
6
Stratum 1
12
Stratum 2
5
Stratum 3
8
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse Events were collected starting with the date of the first dose of study drug until 30 days after last dose of study drug, ongoing related adverse events were continued to be followed until resolution, on average of 3 years..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations. This technology called the "Pediatric Gene Analysis Platform" includes a genomic report (gene expression profile) and a DNA Mutation Panel Report that are being used to discover new ways to understand cancers and potentially predict the best treatments for patients with cancer in the future.
Publications & conference data
4 peer-reviewed publications reference this trial (live from Europe PMC):
NCT07399821 — An Imaging Study of Anti-GD2-800CW in Patients With Neuroblastoma
· Phase 1, PHASE2
· recruiting
NCT06995872 — Phase I Trial of rhIL-15 Plus Dinutuximab Plus Irinotecan/Temozolomide for Children and Young Adults With Relapsed/Refra
· Phase 1
· recruiting
NCT06942039 — Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation
· EARLY_PHASE1
· recruiting
NCT07067346 — Safety & Efficacy of IR-101 in Relapsed/Refractory Neuroblastoma
· EARLY_PHASE1
· recruiting
NCT07027748 — Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or R
· Phase 1
· recruiting
Other Giselle Sholler trials
Trials by the same sponsor.
NCT06540963 — Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
· Phase 2
· recruiting
NCT04301843 — Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma
· Phase 2
· recruiting
NCT02395666 — Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission
· Phase 2
· completed
NCT02162732 — Molecular-Guided Therapy for Childhood Cancer
· NA
· completed
NCT02139397 — Study of Difluoromethylornithine (DFMO) in Combination With Bortezomib for Relapsed or Refractory Neuroblastoma
· Phase 1, PHASE2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Giselle Sholler
Last refreshed: 6 August 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01802567.