# NMTRC003/003B (DFMO maintenance)

Source: https://onco.cc/trials/nmtrc003/  
OnCo record `nmtrc003` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-07
- Tags: lesson:external-control
- Registry id: 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
- Enrolled: 105
- Result: EFS HR 0.48 vs external control.
- Outcomes: Event-free survival: Eflornithine vs External control (ANBL0032), HR 0.48
- Replication: Not replicated in a randomised trial; uptake tracked in ANBL1531.

## Sources

- ClinicalTrials.gov NCT02395666: https://clinicaltrials.gov/study/NCT02395666

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- drugs: [Eflornithine (DFMO)](https://onco.cc/drugs/eflornithine/)
- terms: [Bayesian trial design](https://onco.cc/terms/bayesian-trial-design/), [Clinical equipoise](https://onco.cc/terms/clinical-equipoise/), [External and synthetic control arms](https://onco.cc/terms/external-control-arm/), [Real-world evidence](https://onco.cc/terms/real-world-evidence/), [Single-arm trial](https://onco.cc/terms/single-arm/)
- bottlenecks: [No incentive to repurpose cheap drugs](https://onco.cc/bottlenecks/b-generic-repurposing/), [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)

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