Naxitamab Study 201
Study 201 was the pivotal single-arm study behind naxitamab's approval.
Interim ORR 50% (CR 38%) in 34 evaluable; full-cohort Nature Communications 2025: ORR 50%, median DOR 6.2 months. Outpatient administration.
Setting
Relapsed/refractory high-risk neuroblastoma in bone/bone marrow: naxitamab + GM-CSF
Phase
Phase 2
Sponsor
Y-mAbs
Registry
Headline result
ORR 50%.
Reported
2020
Enrolled
74
In plain words
What these results mean for people, not percentages
Objective response rateprimaryresponse endpoint
- 50 out of 100 people had their tumour shrink with Naxitamab + GM-CSF.
- There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- These results apply to the people the trial enrolled: Relapsed/refractory high-risk neuroblastoma in bone/bone marrow: naxitamab + GM-CSF. People in a different situation may not see the same effect.
- Only 74 people took part, so the numbers are less certain than in a large trial.
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.