# Naxitamab Study 201

Source: https://onco.cc/trials/naxitamab-201/  
OnCo record `naxitamab-201` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Study 201 was the single-arm phase 2 of 74 patients behind naxitamab's approval for relapsed or refractory high-risk neuroblastoma in bone or bone marrow: given with GM-CSF as an outpatient, the antibody produced responses in half of patients. Without a randomised comparison against dinutuximab, equal efficacy is unproven.

## Summary

Naxitamab Study 201, trial NCT03363373 sponsored by Y-mAbs and reported in 2020 with the full cohort published in Nature Communications in 2025, was the pivotal single-arm study behind naxitamab's approval for relapsed or refractory high-risk neuroblastoma in bone or bone marrow. In 74 patients treated with naxitamab and GM-CSF the objective response rate was 50 percent, with complete responses in 38 percent at the interim analysis and durable responses, and the drug can be given as an outpatient. OnCo links it to paediatric neuroblastoma, naxitamab, Nai-Kong V. Cheung and the bottleneck of rare and paediatric cancers without markets. Without a randomised comparison against dinutuximab, whether naxitamab's outpatient convenience comes with equal efficacy is the open question.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-07
- Registry id: NCT03363373
- Phase: 2
- Setting: Relapsed/refractory high-risk neuroblastoma in bone/bone marrow: naxitamab + GM-CSF
- Sponsor: Y-mAbs
- Enrolled: 74
- Result: ORR 50%.
- Outcomes: Objective response rate: Naxitamab + GM-CSF 50%

## Sources

- ClinicalTrials.gov NCT03363373: https://clinicaltrials.gov/study/NCT03363373

## Connected records

- cancers: [High-risk neuroblastoma](https://onco.cc/cancers/neuroblastoma-high-risk/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/)
- technologies: [Monoclonal antibodies](https://onco.cc/technologies/monoclonal-antibody/)
- drugs: [Naxitamab](https://onco.cc/drugs/naxitamab/)
- companies: [Y-mAbs Therapeutics](https://onco.cc/companies/y-mabs/)
- people: [Nai-Kong V. Cheung](https://onco.cc/people/nai-kong-cheung/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/)

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