# ANBL1221

Source: https://onco.cc/trials/anbl1221/  
OnCo record `anbl1221` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

ANBL1221 found that adding the anti-GD2 antibody dinutuximab to irinotecan and temozolomide shrank relapsed neuroblastoma in about half of children, while adding temsirolimus almost never did, and chemo-immunotherapy became the standard salvage treatment.

## Summary

ANBL1221 was a Children's Oncology Group randomised phase 2 selection trial for children with relapsed, refractory or progressive neuroblastoma. Patients received irinotecan and temozolomide with either temsirolimus or dinutuximab plus sargramostim (GM-CSF); the primary endpoint was the objective response rate over the first six cycles. After the randomised part chose dinutuximab, a further 36 patients were enrolled to that arm.

One of 18 patients responded with temsirolimus against 9 of 17 (52.9 percent) with dinutuximab; in all 53 patients treated with irinotecan, temozolomide, dinutuximab and GM-CSF the objective response rate was 41.5 percent with one-year progression-free survival of 67.9 percent and overall survival of 84.9 percent. Pain, fever and infection, neutropenia and hypokalaemia were the common grade 3 or higher toxicities. The regimen is the standard chemo-immunotherapy for relapsed neuroblastoma on the corpus's high-risk neuroblastoma page and was moved into first-line induction in ANBL17P1.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-22
- Also known as: COG ANBL1221
- Tags: soc-trials
- Registry id: NCT01767194
- Phase: 2
- Setting: Children with relapsed, refractory or progressive neuroblastoma: irinotecan and temozolomide with either temsirolimus or the anti-GD2 antibody dinutuximab plus GM-CSF, randomised, then a non-randomised expansion of the dinutuximab arm
- Sponsor: National Cancer Institute (NCI)
- Enrolled: 73
- Result: Objective response 52.9 percent with irinotecan, temozolomide and dinutuximab against 5.6 percent with temsirolimus in the randomised part; 41.5 percent across all 53 dinutuximab-treated patients with one-year overall survival of 84.9 percent.
- Outcomes: Objective response over the first 6 cycles, randomised patients: Irinotecan + temozolomide + dinutuximab + GM-CSF 52.9% vs Irinotecan + temozolomide + temsirolimus 5.6%; Objective response, all patients given dinutuximab (randomised and expansion): Irinotecan + temozolomide + dinutuximab + GM-CSF 41.5%; Progression-free survival at 1 year, all dinutuximab patients: Irinotecan + temozolomide + dinutuximab + GM-CSF 67.9%; Overall survival at 1 year, all dinutuximab patients: Irinotecan + temozolomide + dinutuximab + GM-CSF 84.9%

## Sources

- ClinicalTrials.gov NCT01767194: https://clinicaltrials.gov/study/NCT01767194

## Connected records

- cancers: [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/)
- drugs: [Dinutuximab (ch14.18) / dinutuximab beta](https://onco.cc/drugs/dinutuximab/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Sargramostim](https://onco.cc/drugs/sargramostim/), [Temozolomide](https://onco.cc/drugs/temozolomide/), [Temsirolimus](https://onco.cc/drugs/temsirolimus/)
- companies: [United Therapeutics](https://onco.cc/companies/united-therapeutics/)
- institutions: [Children's Oncology Group (COG)](https://onco.cc/institutions/childrens-oncology-group/)
- key papers: [ANBL1221 expansion: irinotecan, temozolomide and dinutuximab with GM-CSF in refractory or relapsed neuroblastoma](https://onco.cc/key-papers/paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020/), [ANBL1221: irinotecan-temozolomide with temsirolimus or dinutuximab in relapsed or refractory neuroblastoma](https://onco.cc/key-papers/paper-anbl1221-irinotecan-temozolomide-dinutuximab-mody-lancet-oncol-2017/)

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