{"entity":{"id":"nmtrc003","kind":"trial","name":"NMTRC003/003B (DFMO maintenance)","aka":[],"tldr":"NMTRC003 was the single-arm study, compared against historical patients, that got eflornithine approved as maintenance; the design remains debated.","summary":"NMTRC003 and 003B, trial NCT02395666 sponsored by the Beat Childhood Cancer Consortium and published in JCO in 2024, was the single-arm study, compared against historical patients, that got oral eflornithine approved as two years of maintenance for high-risk neuroblastoma in remission after standard therapy including anti-GD2 immunotherapy, and the design remains debated. In 105 enrolled patients, event-free and overall survival were better than in a propensity-matched external control drawn from ANBL0032, and the FDA approved the drug in December 2023 while acknowledging the externally controlled design in a rare paediatric setting. A randomised trial has not been done, and uptake is being tracked in ANBL1531, so whether the benefit is real remains the open question.","status":"positive","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT02395666","url":"https://clinicaltrials.gov/study/NCT02395666"}],"tags":["lesson:external-control"],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":[],"targets":[],"drugs":["eflornithine"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"nct":"NCT02395666","phase":"2","setting":"High-risk neuroblastoma in remission after standard therapy including anti-GD2: 2 years of oral eflornithine (single arm, externally controlled)","sponsor":"Beat Childhood Cancer Consortium","result":"EFS HR 0.48 vs external control.","started":"2015-03-05","startedType":"actual","yearReported":2023,"enrolled":105,"enrolledBasis":"registry","outcomes":[{"endpoint":"Event-free survival","primary":true,"arms":[{"name":"Eflornithine","n":90},{"name":"External control (ANBL0032)","n":270}],"hr":0.48,"ci":[0.27,0.85],"source":"https://ascopubs.org/doi/10.1200/JCO.24.00546"}],"replication":"Not replicated in a randomised trial; uptake tracked in ANBL1531."},"route":"/trials/nmtrc003/","neighbours":{"cancer":[{"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/"}],"drug":[{"id":"eflornithine","kind":"drug","name":"Eflornithine (DFMO)","route":"/drugs/eflornithine/"}],"term":[{"id":"bayesian-trial-design","kind":"term","name":"Bayesian trial design","route":"/terms/bayesian-trial-design/"},{"id":"clinical-equipoise","kind":"term","name":"Clinical equipoise","route":"/terms/clinical-equipoise/"},{"id":"external-control-arm","kind":"term","name":"External and synthetic control arms","route":"/terms/external-control-arm/"},{"id":"real-world-evidence","kind":"term","name":"Real-world evidence","route":"/terms/real-world-evidence/"},{"id":"single-arm","kind":"term","name":"Single-arm trial","route":"/terms/single-arm/"}],"bottleneck":[{"id":"b-generic-repurposing","kind":"bottleneck","name":"No incentive to repurpose cheap drugs","route":"/bottlenecks/b-generic-repurposing/"},{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"}]}}