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NMTRC003 was the single-arm study, compared against historical patients, that got eflornithine approved as maintenance; the design remains debated.
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.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Shares Eflornithine (DFMO), High-risk neuroblastoma, No incentive to repurpose cheap drugs, Neuroblastoma (paediatric).
Shares External and synthetic control arms, Single-arm trial, Real-world evidence.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares No incentive to repurpose cheap drugs, Real-world evidence, Rare and paediatric cancers without markets.
Shares Eflornithine (DFMO), High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.
Shares High-risk neuroblastoma, Neuroblastoma (paediatric), Rare and paediatric cancers without markets.