{"entity":{"id":"anbl1221","kind":"trial","name":"ANBL1221","aka":["COG ANBL1221"],"tldr":"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.\n\nOne 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.","status":"positive","asOf":"2026-09-22","links":[{"label":"ClinicalTrials.gov NCT01767194","url":"https://clinicaltrials.gov/study/NCT01767194"}],"tags":["soc-trials"],"related":[],"cancers":["neuroblastoma-high-risk","neuroblastoma","childhood-cancers"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["irinotecan","temozolomide","dinutuximab","temsirolimus","sargramostim"],"companies":["united-therapeutics"],"institutions":["childrens-oncology-group"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-anbl1221-irinotecan-temozolomide-dinutuximab-mody-lancet-oncol-2017","paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020"],"journals":[],"dependsOn":[],"notes":[],"nct":"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)","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.","yearReported":2017,"enrolled":73,"enrolledBasis":"registry","outcomes":[{"endpoint":"Objective response over the first 6 cycles, randomised patients","primary":true,"unit":"%","arms":[{"name":"Irinotecan + temozolomide + dinutuximab + GM-CSF","n":17,"value":52.9,"note":"95% CI 29.2 to 76.7; 4 partial and 5 complete responses"},{"name":"Irinotecan + temozolomide + temsirolimus","n":18,"value":5.6,"note":"95% CI 0.0 to 16.1"}],"source":"https://doi.org/10.1016/S1470-2045(17)30355-8"},{"endpoint":"Objective response, all patients given dinutuximab (randomised and expansion)","primary":true,"unit":"%","arms":[{"name":"Irinotecan + temozolomide + dinutuximab + GM-CSF","n":53,"value":41.5,"note":"95% CI 28.2 to 54.8; stable disease in a further 22 of 53"}],"source":"https://doi.org/10.1200/JCO.20.00203"},{"endpoint":"Progression-free survival at 1 year, all dinutuximab patients","unit":"%","arms":[{"name":"Irinotecan + temozolomide + dinutuximab + GM-CSF","n":53,"value":67.9}],"source":"https://doi.org/10.1200/JCO.20.00203"},{"endpoint":"Overall survival at 1 year, all dinutuximab patients","unit":"%","arms":[{"name":"Irinotecan + temozolomide + dinutuximab + GM-CSF","n":53,"value":84.9}],"source":"https://doi.org/10.1200/JCO.20.00203"}]},"route":"/trials/anbl1221/","neighbours":{"cancer":[{"id":"childhood-cancers","kind":"cancer","name":"Childhood cancers (all types)","route":"/cancers/childhood-cancers/"},{"id":"neuroblastoma-high-risk","kind":"cancer","name":"High-risk neuroblastoma","route":"/cancers/neuroblastoma-high-risk/"},{"id":"neuroblastoma","kind":"cancer","name":"Neuroblastoma (paediatric)","route":"/cancers/neuroblastoma/"}],"technology":[{"id":"cytotoxic-chemotherapy","kind":"technology","name":"Cytotoxic chemotherapy","route":"/technologies/cytotoxic-chemotherapy/"}],"drug":[{"id":"dinutuximab","kind":"drug","name":"Dinutuximab (ch14.18) / dinutuximab beta","route":"/drugs/dinutuximab/"},{"id":"irinotecan","kind":"drug","name":"Irinotecan (and liposomal irinotecan)","route":"/drugs/irinotecan/"},{"id":"sargramostim","kind":"drug","name":"Sargramostim","route":"/drugs/sargramostim/"},{"id":"temozolomide","kind":"drug","name":"Temozolomide","route":"/drugs/temozolomide/"},{"id":"temsirolimus","kind":"drug","name":"Temsirolimus","route":"/drugs/temsirolimus/"}],"company":[{"id":"united-therapeutics","kind":"company","name":"United Therapeutics","route":"/companies/united-therapeutics/"}],"institution":[{"id":"childrens-oncology-group","kind":"institution","name":"Children's Oncology Group (COG)","route":"/institutions/childrens-oncology-group/"}],"paper":[{"id":"paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020","kind":"paper","name":"ANBL1221 expansion: irinotecan, temozolomide and dinutuximab with GM-CSF in refractory or relapsed neuroblastoma","route":"/key-papers/paper-anbl1221-expansion-dinutuximab-gm-csf-mody-jco-2020/"},{"id":"paper-anbl1221-irinotecan-temozolomide-dinutuximab-mody-lancet-oncol-2017","kind":"paper","name":"ANBL1221: irinotecan-temozolomide with temsirolimus or dinutuximab in relapsed or refractory neuroblastoma","route":"/key-papers/paper-anbl1221-irinotecan-temozolomide-dinutuximab-mody-lancet-oncol-2017/"}]}}