OnCo
trialsTrialPositive

CARTITUDE-1

The study that showed one CAR-T infusion could keep a third of heavily pretreated myeloma patients progression-free for five years without further treatment.

ORR 98%, sCR 83%; median PFS 34.9 months; at ≥5 years, 33% progression-free with no maintenance (JCO 2025), MRD-negative sustained in most. Movement disorders in ~5% (early cohort).

Setting
Relapsed/refractory myeloma after ≥3 lines, triple-class exposed: cilta-cel single infusion
Phase
Phase 1/2
Sponsor
Janssen / Legend
Registry
Headline result
ORR 98%; ~33% progression-free at 5 years without maintenance.
Reported
2021
Enrolled
97

Outcomes

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In plain words
What these results mean for people, not percentages
97 people took part
Objective response rateprimaryresponse endpoint
  • 97.9 out of 100 people had their tumour shrink with Cilta-cel.
  • 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.
Progression-free at 5 yearssurrogate endpoint
  • 33 out of 100 people alive without the cancer growing at 5 years with Cilta-cel.
  • This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
Be careful
  • There was no comparison group, so this number cannot tell you how much of the effect is due to the treatment.
  • These results apply to the people the trial enrolled: Relapsed/refractory myeloma after ≥3 lines, triple-class exposed: cilta-cel single infusion. People in a different situation may not see the same effect.
  • Only 97 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.

97 participants enrolled.

Objective response rateprimary
Cilta-cel97.9 of 100
n = 97
Progression-free at 5 years
Cilta-cel33 of 100
Source
EndpointArmnValueHR (95% CI)pSource
Objective response rateprimaryCilta-cel9797.9%
Progression-free at 5 yearsCilta-cel33%link

Key papers

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Connected

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