TRANSCEND CLL 004
The study that brought CAR-T to CLL: one in five heavily pretreated patients achieved complete remission, most of them lasting.
Primary analysis set (BTKi and venetoclax failure, n=49-50): CR/CRi 18-20%, ORR 42-47%, uMRD in blood 64%; complete responders had durable remissions beyond 2 years. Lancet 2023. Accelerated approval 14 March 2024. Efficacy is lower than in lymphoma, reflecting T-cell dysfunction in CLL.
Setting
Relapsed/refractory CLL/SLL after BTK inhibitor (and venetoclax in the primary analysis set): lisocabtagene maraleucel
Phase
Phase 1/2
Sponsor
BMS / Juno
Registry
Headline result
CR/CRi 18-20%; ORR 47%; uMRD blood 64%.
Reported
2023
Enrolled
137
Replication
Academic CD19 CAR-T series in CLL (Penn, Seattle) report similar CR rates and show ibrutinib co-administration improves T-cell fitness.
In plain words
What these results mean for people, not percentages
Complete response / CRi (primary analysis set)primarysurrogate endpoint
- 18 out of 100 people had no sign of cancer on scans or tests with Liso-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.
Overall response rateresponse endpoint
- 47 out of 100 people had their tumour shrink with Liso-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.
Undetectable MRD in bloodsurrogate endpoint
- 64 out of 100 people had no detectable disease on sensitive tests with Liso-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 CLL/SLL after BTK inhibitor (and venetoclax in the primary analysis set): lisocabtagene maraleucel. People in a different situation may not see the same effect.
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.
137 participants enrolled.
Overall response rate
Liso-cel47 of 100
Undetectable MRD in blood
Liso-cel64 of 100
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Complete response / CRi (primary analysis set)primary | Liso-cel | 49 | 18% | — | — | link |
| Overall response rate | Liso-cel | — | 47% | — | — | — |
| Undetectable MRD in blood | Liso-cel | — | 64% | — | — | — |
Replication
Academic CD19 CAR-T series in CLL (Penn, Seattle) report similar CR rates and show ibrutinib co-administration improves T-cell fitness.