NCT04385277|Active Not RecruitingPhase 2ResultsFDA Drug

Treatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL)

A Pilot Study of Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide in the Post-Consolidation Setting for High-Risk Neuroblastoma

Children's Oncology Group ClinicalTrials.gov

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3 other identifiers

ANBL19P1NCI-2020-02950U10CA180886

Study Type

interventional

Target

Locations

4 countries

Sites

Timeline

RegisteredMay 2020

StartedDec 2020

CompletionSep 2027

Brief Summary

This phase II trial studies if dinutuximab, GM-CSF, isotretinoin in combination with irinotecan, and temozolomide (chemo-immunotherapy) can be given safely to patients with high-risk neuroblastoma after Consolidation therapy (which usually consists of two autologous stem cell transplants and radiation) who have not experienced worsening or recurrence of their disease. Dinutuximab represents a kind of cancer therapy called immunotherapy. Unlike chemotherapy and radiation, dinutuximab targets the cancer cells without destroying nearby healthy cells. Sargramostim helps the body produce normal infection-fighting white blood cells. Isotretinoin helps the neuroblastoma cells become more mature. These 3 drugs (standard immunotherapy) are already given to patients with high-risk neuroblastoma after Consolidation because they have been proven to be beneficial in this setting. Chemotherapy drugs, such as irinotecan and temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. They may also affect how well immunotherapy works on neuroblastoma cells. Giving chemo-immunotherapy after intensive therapy may work better in treating patients with high-risk neuroblastoma compared to standard immunotherapy.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P25-P50 for phase_2

Timeline

17mo left

Started Dec 2020

Longer than P75 for phase_2

Geographic Reach

4 countries

77 active sites

Status

active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

6.8 years study duration

Study Progress79%

Dec 2020Sep 2027

First Submitted

Initial submission to the registry

May 8, 2020

Completed

4 days until next milestone

First Posted

Study publicly available on registry

May 12, 2020

Completed

8 months until next milestone

Study Start

First participant enrolled

December 31, 2020

Completed

2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2023

Completed

1.1 years until next milestone

Results Posted

Study results publicly available

August 14, 2024

Completed

3.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2027

Expected

Last Updated

October 27, 2025

Status Verified

October 1, 2025

Enrollment Period

2.5 years

First QC Date

May 8, 2020

Results QC Date

May 28, 2024

Last Update Submit

October 23, 2025

Conditions

Ganglioneuroblastoma, Nodular Neuroblastoma

Outcome Measures

Primary Outcomes (1)

Percentage of Patients Who Complete 5 Cycles of Dinutuximab + Chemotherapy Without Progressive Disease (PD)
Will be assessed by estimation of the feasibility therapy completion rate together with a 95% Wilson confidence interval (CI).
Within 30 weeks from the date of first treatment

Secondary Outcomes (2)

Event-free Survival (EFS)
From the time of start of protocol therapy to the occurrence of disease relapse or progression, secondary malignancy, or death, assessed at 1 year.
Overall Survival (OS)
From the time of start of protocol therapy to death, assessed at 1 year.

Study Arms (1)

Treatment (temozolomide, irinotecan, dinutuximab)

EXPERIMENTAL

Patients receive temozolomide PO or via enteral tube daily and irinotecan IV over 90 minutes daily on days 1-5, dinutuximab IV over 10-20 hours daily on days 2-5, sargramostim SC or IV over 2 hours daily on days 6-12, and isotretinoin PO BID on days 8-21. Patients undergo MUGA during screening. Patients also undergo MRI, or CT, I23I-MIBG, or FDG-PET, BM aspiration, and BM biopsy on study. Treatment repeats every 28 days for up to 5 cycles (up to 6 cycles for isotretinoin only) in the absence of disease progression or unacceptable toxicity.

Procedure: Biospecimen CollectionProcedure: Bone Marrow AspirationProcedure: Bone Marrow BiopsyProcedure: Computed TomographyBiological: DinutuximabProcedure: FDG-Positron Emission TomographyRadiation: Iobenguane I-123Drug: IrinotecanDrug: IsotretinoinProcedure: Magnetic Resonance ImagingProcedure: Multigated Acquisition ScanBiological: SargramostimDrug: Temozolomide